| Publication Title | Journal | Authors | Pub Date | PubMed | Project PIs |
|---|---|---|---|---|---|
| AAV for gene therapy drives a nephrotoxic response via NFκB in kidney organoids | Signal Transduction and Targeted Therapy | Gupta et al. | 8/8/25 | 40774951 | Morizane, Lewis, Sabbisetti |
| GFP-on mouse model for interrogation of in vivo gene editing | Nature Communications | Dib et al. | 7/31/25 | 40744920 | Liu |
| Focused ultrasound-mediated APOE4 knockdown in mouse brain | Alzheimers Dementia | Zheng et al. | 7/22/25 | 40665471 | Leong |
| Variable Neuronal Expression of Green Florescent Protein in the Brain of a Transgenic Swine Reporter Model | Next Research | Hower et al. | 7/16/25 | 40800570 | Gong, Emborg, Levine, Roy, Saha |
| Brain endothelial gap junction coupling enables rapid vasodilation propagation during neurovascular coupling | Cell | Krolak et al. | 7/11/25 | 40675149 | Deverman |
| Systemic in utero gene editing as a treatment for cystic fibrosis | Proceedings of the National Academy of Sciences | Ricciardi et al. | 6/17/25 | 40493185 | Saltzman, Glazer |
| Identifying optimal conditions for precise knock-in of exogenous DNA into the zebrafish genome | Development | Oikemus et al. | 6/15/25 | 40446205 | Sontheimer, Khvorova, Wolfe, Watts |
| Multiplex base editing to protect from CD33 directed drugs for immune and gene therapy | Nature Communications | Borot et al. | 6/2/25 | 40425554 | Liu |
| High-purity AAV vector production utilizing recombination-dependent minicircle formation and genetic coupling | EMBO molecular medicine | Liu et al. | 5/16/25 | 40379974 | Gao, Anderson, Xue |
| Self-assembling protein nanoparticles for cytosolic delivery of nucleic acids and proteins | Nature biotechnology | Eweje et al. | 5/15/25 | 40374955 | Chaikof |
| Precision enhancement of CAR-NK cells through non-viral engineering and highly multiplexed base editing | Journal for immunotherapy of cancer | Wang et al. | 5/7/25 | 40341025 | Carlson |
| Spillover can limit accurate signal quantification in MPI | NPJ Imaging | Shakeri-Zadeh et al. | 5/6/25 | 40604257 | Bulte |
| GAA replacement improves respiratory muscle, neural, and alveolar pathology in the pompe mouse | Respiratory physiology and neurobiology | Roger et al. | 4/25/25 | 40288624 | Asokan |
| Rapid two-step target capture ensures efficient CRISPR-Cas9-guided genome editing | Molecular cell | Shi et al. | 4/23/25 | 40273916 | Doudna, Banfield |
| Deletion of Exon 51 in a Humanized Duchenne Muscular Dystrophy Mouse Model Abolishes Ventricular Arrhythmia Predisposition | Circulation | Perelli et al. | 4/10/25 | 40207359 | Asokan |
| Gene regulation technologies for gene and cell therapy | Molecular therapy | Butterfield et al. | 4/6/25 | 40195118 | Gersbach |
| Dynamic basis of supercoiling-dependent DNA interrogation by Cas12a via R-loop intermediates | Nature Communications | Aris et al. | 3/26/25 | 40133266 | Doudna, Banfield |
| Human Myobundle Platform for Studying the Role of Notch Signaling in Satellite Cell Phenotype and Function | Advanced healthcare Materials | Broer et al. | 3/24/25 | 40123310 | Gersbach, Bursac, Truskey |
| A deep learning model trained on expressed transcripts across different tissue types reveals cell-type codon-optimization preferences | Nucleic Acids Research | Ravi et al. | 3/20/25 | 40156867 | Gao, Anderson, Xue |
| Characterization of diverse Cas9 orthologs for genome and epigenome editing | Proceedings of the National Academy of Sciences | Butterfield et al. | 3/12/25 | 40073054 | Gersbach |
| Investigation of the protein corona and biodistribution profile of polymeric nanoparticles for intra-amniotic delivery | Biomaterials | Lynn et al. | 3/6/25 | 40064138 | Saltzman, Glazer |
| Peptide-enabled ribonucleoprotein delivery for CRISPR engineering (PERC) in primary human immune cells and hematopoietic stem cells | Nature Protocols | Sahu et al. | 3/3/25 | 40032999 | Wilson, Doudna |
| Improving adenine base editing precision by enlarging the recognition domain of CRISPR-Cas9 | Nature Communications | Gao et al. | 2/28/25 | 40021632 | Chen, Liu |
| The reverse transcriptase domain of prime editors contributes to DNA repair in mammalian cells | Nature Biotechnology | Zheng et al. | 2/17/25 | 39962280 | Gao, Anderson, Xue |
| Activation of the imprinted Prader-Willi syndrome locus by CRISPR-based epigenome editing | Cell genomics | Rohm et al. | 2/12/25 | 39947136 | Gersbach |
| MBNL overexpression rescues cardiac phenotypes in a myotonic dystrophy type 1 heart mouse model | The Journal of Clinical Investigation | Hu et al. | 2/11/25 | 39932794 | Heaney, Dickinson, Lagor |
| Evolving adeno-associated viruses for gene transfer to the kidney via cross-species cycling of capsid libraries | Nature biomedical engineering | Rosales et al. | 2/5/25 | 39910375 | Asokan |
| MPI performance of magnetic nanoparticles depends on matrix composition and temperature: implications for in vivo MPI signal amplitude, spatial resolution, and tracer quantification | Nanoscale advances | Salimi et al. | 1/15/25 | 39906142 | Bulte |
| CRISPR-Cas12a bends DNA to destabilize base pairs during target interrogation | Nucleic acids research | Soczek et al. | 1/11/25 | 39698811 | Doudna |
| Functional protein mining with conformal guarantees | Nature Communications | Boger et al. | 1/2/25 | 39747192 | Doudna |
| Do Pulmonary Ionocytes Absorb Chloride or Secrete Chloride? | American Journal of Physiology | Thornell et al. | 12/23/24 | 39716827 | McCray |
| Advances in acid-degradable and enzyme-cleavable linkers for drug delivery | Current opinion in Chemical Biology | Zhao et al. | 12/5/24 | 39642424 | Bankiewicz, Murthy |
| Osteosarcoma: A comprehensive review of model systems and experimental therapies | Medical research archives | Robbins et al. | 11/29/24 | 39916749 | Carlson |
| Safer and efficient base editing and prime editing via ribonucleoproteins delivered through optimized lipid-nanoparticle formulations | Nature biomedical engineering | Holubowicz et al. | 11/28/24 | 39609561 | Liu |
| Ungated, plug-and-play preclinical cardiac CEST-MRI using radial FLASH with segmented saturation | Magnetic Resonance in Medicine | Weigand-Whittier et al. | 11/28/24 | 39607872 | Vandsburger |
| Lipid nanoparticle-mediated mRNA delivery to CD34+ cells in rhesus monkeys | Nature Biotechnology | Kim et al. | 11/22/24 | 39578569 | Tarantal, Hartigan-O'Connor, Segal |
| In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice | Proceedings of the National Academy of Sciences | Du et al. | 11/18/24 | 39556729 | Liu |
| Directed evolution of engineered virus-like particles with improved production and transduction efficiencies | Nature Biotechnology | Raguram et al. | 11/13/24 | 39537813 | Liu |
| In vitro vascularization improves in vivo functionality of human engineered cardiac tissues | Acta biomaterialia | Li et al. | 11/10/24 | 39528062 | Gersbach, Bursac, Truskey |
| Engineered Cardiac Tissues as a Platform for CRISPR-Based Mitogen Discovery | Advanced healthcare Materials | DeLuca et al. | 11/7/24 | 39508305 | Gersbach, Bursac, Truskey |
| Engineered Cas9 variants bypass Keap1-mediated degradation in human cells and enhance epigenome editing efficiency | Nucleic acids research | Chen et al. | 10/28/24 | 39228373 | Gersbach, Bursac, Truskey |
| Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review | Molecular therapy | Wang et al. | 10/28/24 | 39489915 | Gao, Anderson, Xue |
| Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs | Human gene Therapy | Castillo et al. | 9/24/24 | 39212664 | Clark, Ekker |
| Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiency | JCI Insight | Du et al. | 10/8/24 | 39137042 | Gao, Anderson, Xue |
| Increasing intracellular dNTP levels improves prime editing efficiency | Nature biotechnology | Liu et al. | 9/25/24 | 39322763 | Sontheimer, Khvorova, Wolfe, Watts |
| Amphiphilic shuttle peptide delivers base editor ribonucleoprotein to correct the CFTR R553X mutation in well-differentiated airway epithelial cells | Nucleic acids research | Kulhankova et al. | 9/24/24 | 39315713 | Liu |
| Engineered myovascular tissues for studies of endothelial/satellite cell interactions | Acta biomaterialia | Broer et al. | 9/19/24 | 39299621 | Gersbach, Bursac, Truskey |
| Tuning Lipid Nanoparticles for RNA Delivery to Extrahepatic Organs | Advanced materials | Song et al. | 9/5/24 | 39233550 | Chen, Liu |
| Animal and bacterial viruses share conserved mechanisms of immune evasion | Cell | Hobbs et al. | 8/27/24 | 39197447 | Doudna, Banfield | Wilson |
| Acid-degradable lipid nanoparticles enhance the delivery of mRNA | Nature nanotechnology | Zhao et al. | 8/23/24 | 39179796 | Bankiewicz, Murthy |
| The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2 | Nature Gene Therapy | Cui et al. | 8/12/24 | 39134629 | Gao, Anderson, Xue |
| T Cell Responses during Human Immunodeficiency Virus/ Mycobacterium tuberculosis Coinfection | Vaccines | Bohorquez et al. | 8/9/24 | 39204027 | Yi |
| Next generation triplex-forming PNAs for site-specific genome editing of the F508del CFTR mutation | Journal of cystic fibrosis | Gupta et al. | 8/5/24 | 39107154 | Glazer, Ly, Saltzman |
| Systematic multi-trait AAV capsid engineering for efficient gene delivery | Nature Communications | Eid et al. | 8/4/24 | 39097583 | Deverman |
| Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease | Molecular therapy | Katta et al. | 7/31/24 | 39086133 | Tsai |
| Epigenome editing technologies for discovery and medicine | Nature Biotechnology | McCutcheon et al. | 7/29/24 | 39075148 | Gersbach |
| Peptide Nucleic Acid-Mediated Regulation of CRISPR-Cas9 Specificity | Nucleic Acid Therapeutics | Carufe et al. | 7/22/24 | 39037032 | Glazer, Ly, Saltzman |
| Cryopreservation of human kidney organoids | Cellular and molecular life sciences | Mashouf et al. | 7/18/24 | 39023560 | Morizane, Lewis, Sabbisetti |
| Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells | Nature biomedical engineering | Sousa et al. | 7/10/24 | 38987629 | McCray |
| Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation | Science translational Medicine | Zhu et al. | 7/10/24 | 38985856 | Chen, Liu |
| AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys | Human Gene Therapy | Liang et al. | 7/3/24 | 38767512 | Gao, Xue, Anderson | Tarantal, Hartigan-O'Connor, Segal |
| Engineered IgM and IgG cleaving enzymes for mitigating antibody neutralization and complement activation in AAV gene transfer | Molecular therapy | Smith et al. | 7/3/24 | 38715362 | Tarantal, Hartigan-O'Connor, Segal |
| Branching in poly(amine-co-ester) polyplexes impacts mRNA transfection | Science translational Medicine | Shin et al. | 6/27/24 | 38986360 | Saltzman, Glazer |
| Engineering a targeted and safe bone anabolic gene therapy to treat osteoporosis in alveolar bone loss | Molecular Therapy: The Journal of the American Society of Gene Therapy | Lin et al. | 6/26/24 | 38937970 | Glazer, Ly, Saltzman |
| Bioengineered Model of Human LGMD2B Skeletal Muscle Reveals Roles of Intracellular Calcium Overload in Contractile and Metabolic Dysfunction in Dysferlinopathy | Advanced science | Khodabukus et al. | 6/17/24 | 38887849 | Gersbach, Bursac, Truskey |
| Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing | Nature Biomedical Engineering | Pandey et al. | 6/10/24 | 38858586 | Liu |
| Evolution of the clinical-stage hyperactive TcBuster transposase as a platform for robust non-viral production of adoptive cellular therapies | Molecular Therapy | Skeate el a. | 6/5/24 | 38627969 | Carlson |
| Advances and challenges in modeling inherited peripheral neuropathies using iPSCs | Experimental & molecular medicine | Lent et al. | 6/3/24 | 38825644 | Conklin |
| Multiscale engineering of brain organoids for disease modeling | Advanced drug Delivery Reviews | Xu et al. | 5/27/24 | 38810702 | Leong |
| Modifying the Backbone Chemistry of PEG-Based Bottlebrush Block Copolymers for the Formation of Long-Circulating Nanoparticles | Advanced healthcare Materials | Grundler et al. | 5/22/24 | 38734871 | Glazer, Ly, Saltzman |
| AAV5 delivery of CRISPR/Cas9 mediates genome editing in the lungs of young rhesus monkeys | Human Gene Therapy | Xue et al. | 5/20/24 | 38767512 | Gao, Anderson, Xue | Tarantal |
| Enhancing in vivo cell and tissue targeting by modulation of polymer nanoparticles and macrophage decoys | Nature Communications | Piotrowski-Daspit et al. | 5/18/24 | 38762483 | Saltzman, Glazer |
| An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery | Science | Huang et al. | 5/16/24 | 38753766 | Deverman |
| Modifying the Backbone Chemistry of PEG-Based Bottlebrush Block Copolymers for the Formation of Long-Circulating Nanoparticles | Advanced healthcare Materials | Grundler et al. | 5/11/24 | 38734871 | Glazer, Ly, Saltzman |
| A novel humanized mouse model for HIV and tuberculosis co-infection studies | Frontiers in Immunology | Bohorquez et al. | 5/10/24 | 38799434 | Yi |
| Imaging CAR-NK cells targeted to HER2 ovarian cancer with human sodium-iodide symporter-based positron emission tomography | European Journal of Nuclear Medicine and Molecular Imaging | Shalaby et al. | 5/9/24 | 38722382 | Ronald |
| Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus | Human Gene Therapy | Sato et al. | 5/3/24 | 38661537 | Gao, Anderson, Xue |
| Imaging of adeno-associated viral capsids for purposes of gene editing using CEST NMR/MRI | Magnetic Resonance in Medicine | Lam et al. | 4/23/24 | 38651648 | Vandsburger |
| Advancing preclinical drug evaluation through automated 3D imaging for high-throughput screening with kidney organoids | Biofabrication | Oishi et al. | 8/20/24 | 38547531 | Morizane, Lewis, Sabbisetti |
| Adeno-associated virus as a delivery vector for gene therapy of human diseases | Signal Transduction and Targeted Therapy | Wang et al. | 4/3/24 | 38565561 | Gao, Anderson, Xue |
| High-throughput evaluation of genetic variants with prime editing sensor libraries | Nature Biotechnology | Gould et al. | 3/12/24 | 38472508 | Liu |
| Bacteriophage therapy for the treatment of Mycobacterium tuberculosis infections in humanized mice | Communications Biology | Yang et al. | 3/9/24 | 38461214 | Yi |
| Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy | Nature Biomedical Engineering | Alves et al. | 3/8/24 | 38057426 | Tsai |
| Targeted nonviral delivery of genome editors in vivo | Proceedings of the National Academy of Sciences | Tsuchida et al. | 3/4/24 | 38437567 | Doudna, Banfield |
| Cationic cholesterol-dependent LNP delivery to lung stem cells, the liver, and heart | Proceedings of the National Academy of Sciences | Radmand et al. | 3/4/24 | 38437539 | Dahlman, Santangelo |
| A humanized mouse model for adeno-associated viral gene therapy | Nature Communications | Barzi et al. | 3/4/24 | 38438373 | Gersbach, Bursac, Truskey |
| Compartmentalized ocular lymphatic system mediates eye-brain immunity | Nature | Yin et al. | 2/28/24 | 38418880 | Saltzman, Glazer |
| Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference | Nature Communications | Zhang et al. | 2/24/24 | 38402281 | Liu |
| Efficient prime editing in two-cell mouse embryos using PEmbryo | Nature Biotechnology | Kim-Yip et al. | 2/6/24 | 38321114 | Liu |
| Advancements in therapeutic development: kidney organoids and organs on a chip | Kidney International | Tabibzadeh and Morizane | 1/29/24 | 38296026 | Morizane, Lewis, Sabbisetti |
| Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo | Nucleic acids research | Zhang et al. | 1/25/24 | 38033325 | Liu | Sontheimer, Khvorova, Watts, Wolfe |
| CRISPR Activation Reverses Haploinsufficiency and Functional Deficits Caused by TTN Truncation Variants | Circulation | Ghahremani et al. | 1/18/24 | 38235591 | Hinson |
| TTN truncation variants produce sarcomere-integrating proteins of uncertain functional significance | The journal of Clinical Investigation | Hinson & Campbell | 1/16/24 | 38226618 | Hinson |
| In vivo human T cell engineering with enveloped delivery vehicles | Nature Biotechnology | Hamilton et al. | 1/11/24 | 38212493 | Doudna, Banfield |
| Harnessing non-Watson-Crick's base pairing to enhance CRISPR effectors cleavage activities and enable gene editing in mammalian cells | Proceedings of the National Academy of Sciences | Gao et al. | 1/9/24 | 38150477 | Chen, Liu, Xu |
| Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo | Nature Biotechnology | An et al. | 1/8/24 | 38191664 | Liu |
| Protein-based nanoparticles for therapeutic nucleic acid delivery | Biomaterials | Eweje et al. | 1/2/24 | 38181574 | Chaikof |
| Tumor-tropic Trojan horses: Using mesenchymal stem cells as cellular nanotheranostics | Theranostics | Rosu et al. | 1/1/24 | 38169524 | Bulte |
| AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfecta | Molecular Therapy Nucleic Acids | Yang et al. | 12/26/23 | 38261950 | Gao, Anderson, Xue |
| Eukaryotic RNA-guided endonucleases evolved from a unique clade of bacterial enzymes | Nucleic Acid Research | Yoon et al. | 12/11/23 | 37971304 | Doudna, Banfield |
| Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo | Nature Communications | Kulhankova et al. | 12/5/23 | 38052872 | McCray | Liu | Tarantal |
| Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo | Nucleic Acid Research | Zhang et al. | 11/30/23 | 38033325 | Sontheimer, Khvorova, Watts, Wolfe | Liu |
| Combinatorial development of nebulized mRNA delivery formulations for the lungs | Nature Nanotechnology | Jiang et al. | 11/20/23 | 37985700 | Gao, Anderson, Xue |
| Assigning functionality to cysteines by base editing of cancer dependency genes | Nature Chemical Biology | Li et al. | 11/19/23 | 37783940 | Liu |
| Transcriptional and epigenetic regulators of human CD8+ T cell function identified through orthogonal CRISPR screens | Nature Genetics | McCutcheon et al. | 11/9/23 | 37945901 | Gersbach |
| CasPEDIA Database: a functional classification system for class 2 CRISPR-Cas enzymes | Nucleic Acid Research | Adler et al. | 10/27/23 | 37889041 | Doudna, Banfield |
| DNA recognition and induced genomemodification by a hydroxymethyl-g tail-clamppeptide nucleic acid | Cell Reports Physical Science | Oyaghire et al. | 10/18/23 | 37920723 | Glazer, Ly, Saltzman |
| Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells | Cell | Tsuchida et al. | 10/12/23 | 37794590 | Doudna, Banfield |
| Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery | Nature Protocols | Gonzalez et al. | 9/21/23 | 37735235 | Asokan |
| Lipid Nanoparticles Deliver mRNA to the Brain after an Intracerebral Injection | Biochemistry | Tuma et al. | 9/20/23 | 37729550 | Doudna | Bankiewicz, Murthy |
| Targeted genome editing with a DNA-dependent DNA polymerase and exogenous DNA-containing templates | Nature Biotechnology | Liu et al. | 9/14/23 | 37709915 | Sontheimer, Khvorova, Watts, Wolfe | Xue |
| Phage-assisted evolution and protein engineering yield compact, efficient prime editors | Cell | Doman et al. | 8/31/21 | 37657419 | Liu |
| Polymer nanoparticles deliver mRNA to the lung for mucosal vaccination | Science Translational Medicine | Suberi t al. | 8/16/23 | 37585505 | Saltzman, Glazer |
| Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo | Nature Communications | Tao et al. | 8/15/23 | 37582836 | Chen, Liu, Xu | Liu |
| Focused ultrasound-mediated brain genome editing | Proceedings of the National Academy of Sciences | Lao et al. | 8/14/23 | 37579143 | Leong | Murray, Lutz |
| Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy | The Journal of Clinical Investigation | Kabra et al. | 8/10/23 | 37561581 | Gong, Emborg, Levine, Roy, Saha | Liu |
| Rational nanoparticle design: Optimization using insights from experiments and mathematical models | Journal of Controlled Release | Richfield et al. | 7/22/23 | 37442201 | Saltzman, Glazer |
| Therapeutic in vivo delivery of gene editing agents | Cell | Raguram et al. | 7/21/23 | 35798006 | Liu |
| Reducing the inherent auto-inhibitory interaction within the pegRNA enhances prime editing efficiency | Nucleic Acids Research | Ponnienselvan et al. | 7/21/23 | 37246708 | Sontheimer, Khvorova, Watts, Wolfe |
| Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier | PLOS Biology | Huang et al. | 7/19/23 | 37467291 | Deverman | Heaney, Dickinson, Lagor |
| Potent and uniform fetal hemoglobin induction via base editing | Nature Genetics | Mayuranathan et al. | 7/3/23 | 37400614 | Liu | Tsai |
| Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells | Molecular Therapy Nucleic Acids | Joynt et al. | 6/29/23 | 37547293 | Liu |
| Adenine transversion editors enable precise, efficient A•T-to-C•G base editing in mammalian cells and embryos | Nature Biotechnology | Chen et al. | 6/15/23 | 37322276 | Liu |
| Template-jumping prime editing enables large insertion and exon rewriting in vivo | Nature Communications | Du et al. | 6/8/23 | 37291100 | Gao, Anderson, Xue | Sontheimer |
| Liver directed adeno-associated viral vectors to treat metabolic disease | Journal of inherited Metabolic Disease | Chuecos & Lagor | 6/5/23 | 37254440 | Heaney, Dickinson, Lagor |
| Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8 | Molecular Therapy | Du et al. | 5/22/23 | 37244253 | Chen, Liu, Xu |
| Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes | Nature Biomedical Engineering | Foss et al. | 5/7/23 | 37147433 | Wilson, Doudna |
| A Multifunctional and Highly Adaptable Reporter System for CRISPR/Cas Editing | International Journal of Molecular Sciences | Wettengel et al. | 5/4/23 | 37175977 | Hennebold |
| Efficient prime editing in mouse brain, liver and heart with dual AAVs | Nature Biotechnology | Davis et al. | 5/4/23 | 37142705 | Liu |
| Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice | Nature Biomedical Engineering | Liu et al. | 4/17/23 | 37069266 | Liu |
| Reprogramming by drug-like molecules leads to regeneration of cochlear hair cell-like cells in adult mice | Proceedings of the National Academy of Sciences | Chen et al. | 4/17/23 | 37068229 | Chen, Liu, Xu |
| Base editing rescue of spinal muscular atrophy in cells and in mice | Science | Arbab et al. | 3/30/2023 | 36996170 | Liu | Gao |
| Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing | Nature Biotechnology | Li et al. | 3/30/2023 | 36997680 | Gao, Anderson, Xue |
| Strategies for Improving Vascularization in Kidney Organoids: A Review of Current Trends | Biology | Konoe and Morizane | 3/26/23 | 37106704 | Morizane, Lewis, Sabbisetti |
| CaMKIIδ gene editing- A base hit for the heart | Journal of Cardiovascular Aging | Walkey and Lagor | 3/22/2023 | 37065676 | Heaney, Dickinson, Lagor |
| Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities | International Journal of Molecular Sciences | Kar et al. | 3/18/2023 | 36982871 | Ekker, Clark |
| Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice | Nature Medicine | Reichart et al. | 2/16/2023 | 36797483 | Liu |
| Phenylboronic Acid-Functionalized Polyplexes Tailored to Oral CRISPR Delivery | Nano Letters | Yoshinaga et al. | 2/8/2023 | 36648291 | Leong |
| The Transcriptional Response to Lung-Targeting Lipid Nanoparticles in Vivo | Nano Letters | Radmand et al. | 2/8/2023 | 36701517 | Dahlman, Santangelo |
| Functional maturation of kidney organoid tubules: Piezo1-mediated Ca2+ signaling | American Journal of Physiology - Cell Physiology | Carrisoza-Gaytan et al. | 2/6/2023 | 36745528 | Morizane, Lewis, Sabbisetti |
| In vivo editing of the pan-endothelium by immunity evading simian adenoviral vector | Biomedicine & Pharmacotherapy | Lorincz et al. | 2/1/2023 | 36587560 | Heaney, Dickinson, Lagor | Curiel |
| Focused ultrasound-mediated brain genome editing | Research Square | Lao et al. | 1/20/2023 | 36712096 | Leong | Murray, Lutz |
| Synthetic Biology Design as a Paradigm Shift toward Manufacturing Affordable Adeno-Associated Virus Gene Therapies | ACS Synthetic Biology | Collins et al. | 1/20/2023 | 36627108 | Curiel |
| Reframing the perception of outliers and negative data in translational research | Brain Research Bulletin | Marina E. Emborg | 1/19/2023 | 36464129 | Gong, Emborg, Levine, Roy, Saha |
| Clinical progress in genome-editing technology and in vivo delivery techniques | Trends in Genetics | Jennifer Khirallah et al. | 1/18/2023 | 36669950 | Chen, Liu, Xu |
| Accounting for diversity in the design of CRISPR-based therapeutic genome editing | Nature Genetics | Krishanu Saha | 1/2/2023 | 36593304 | Saha, Gamm, Roy, Skala |
| Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteins | Biomaterials | Metzger et al. | 12/13/2022 | 36527789 | Gong, Emborg, Levine, Roy, Saha | Murray, Lutz |
| In Vivo Cellular Magnetic Imaging: Labeled vs. Unlabeled Cells | Advanced Functional Materials | Bulte et al. | 12/9/2022 | 36589903 | Bulte |
| Substituting racemic ionizable lipids with stereopure ionizable lipids can increase mRNA delivery | Journal of Controlled Release | Da Silva Sanchez et al. | 11/30/2022 | 36423872 | Dahlman, Santangelo |
| Diverse virus-encoded CRISPR-Cas systems include streamlined genome editors | Cell | Al-Sheyab et al. | 11/23/2022 | 36423580 | Doudna, Banfield |
| Compact zinc finger base editors that edit mitochondrial or nuclear DNA in vitro and in vivo | Nature Communications | Willis et al. | 11/23/2022 | 36418298 | Liu |
| Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity | Nature Biotechnology | Neugebauer et al. | 11/10/2022 | 36357719 | Liu |
| Adult human kidney organoids originate from CD24+ cells and represent an advanced model for adult polycystic kidney disease | Nature Genetics | Xu et al. | 10/27/2022 | 36303074 | Freedman |
| Unadjuvanted intranasal spike vaccine elicits protective mucosal immunity against sarbecoviruses | Science | Mao et al. | 10/27/2022 | 36302057 | Saltzman, Glazer |
| Nucleic Acid Delivery to the Vascular Endothelium | Moleculare Pharmaceutics | Reschke et al. | 10/17/2022 | 36251765 | Saltzman, Glazer |
| Noninvasively differentiating acute and chronic nephropathies via multiparametric urea-CEST, nuclear Overhauser enhancement-CEST, and quantitative magnetization transfer MRI | Magnetic Resonance in Medicine | Shin et al. | 10/13/2022 | 36226662 | Vandsburger |
| LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study | Molecular Therapy Methods & Clinical Development | Doerfler et al. | 10/13/2022 | 36381302 | Bao, Lagor |
| Translating musculoskeletal bioengineering into tissue regeneration therapies | Science Translational Medicine | Khodabukus et al. | 10/12/2022 | 36223445 | Gerbach, Bursac, Truskey |
| EZ Clear for simple, rapid, and robust mouse whole organ clearing | eLife | Hsu et al. | 10/11/2022 | 36218247 | Heaney, Dickinson, Lagor |
| Monobody adapter for functional antibody display on nanoparticles for adaptable targeted delivery applications | Nature Communications | Albert et al. | 10/11/2022 | 36220817 | Glazer, Ly, Saltzman |
| Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing | Nature Communications | Gonzalez et al. | 10/10/2022 | 36210364 | Asokan |
| Organoid-on-a-chip model of human ARPKD reveals mechanosensing pathomechanisms for drug discovery | Science Advances | Hiratsuka et al. | 9/23/2022 | 36129975 | Morizane, Lewis, Sabbisetti |
| High-throughput continuous evolution of compact Cas9 variants targeting single-nucleotide-pyrimidine PAMs | Nature Biotechnology | Huang et al. | 9/8/2022 | 36076084 | Liu |
| Efficient Homology-Directed Repair with Circular Single-Stranded DNA Donors | The CRISPR Journal | Iyer et al. | 9/7/2022 | 36070530 | Sontheimer, Khvorova, Watts, Wolfe |
| Promoting validation and cross-phylogenetic integration in model organism research | Disease Models & Mechanisms | Cheng et al. | 9/1/2022 | 36125045 | Heaney, Dickinson, Lagor | Ekker, Clark | Murray, Lutz |
| An ELISA-based platform for rapid identification of structure-dependent nucleic acid-protein interactions detects novel DNA triplex interactors | Journal of Biological Chemistry | Economos et al. | 8/18/2022 | 35988651 | Glazer, Ly, Saltzman |
| Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivo | Nature Communications | Ni et al. | 8/15/2022 | 35970837 | Dahlman, Santangelo |
| Designing and executing prime editing experiments in mammalian cells | Nature Protocols | Doman et al. | 8/8/2022 | 35941224 | Liu |
| Multiomics characterization of mouse hepatoblastoma identifies yes-associated protein 1 target genes | The American Association for the Study of Liver Diseases | Rodríguez et al. | 8/6/2022 | 35932276 | Gao, Anderson, Xue | Sontheimer, Khvorova, Watts, Wolfe |
| Development of 68 Ga-Labeled Hepatitis E Virus Nanoparticles for Targeted Drug Delivery and Diagnostics with PET | Molecular Pharmaceutics | Lambidis et al. | 8/1/2022 | 35857429 | Lam, Cheng |
| Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors | Nature Biomedical Engineering | Davis et al. | 7/28/2022 | 35902773 | Liu | Chaikof |
| Therapeutic in vivo delivery of gene editing agents | Cell | Raguram et al. | 7/21/2022 | 35798006 | Liu |
| Nanoparticle single-cell multiomic readouts reveal that cell heterogeneity influences lipid nanoparticle-mediated messenger RNA delivery | Nature Nanotechnology | Dobrowolski et al. | 6/30/2022 | 35768613 | Dahlman, Santangelo |
| Efficient Genome Editing Achieved via Plug-and-Play Adenovirus Piggyback Transport of Cas9/gRNA Complex on Viral Capsid Surface | ACS Nano | Lu et al. | 6/24/2022 | 35749339 | Curiel |
| Technologies to Assess Drug Response and Heterogeneity in Patient-Derived Cancer Organoids | Annual Review of Biomedical Engineering | Skala et al. | 6/6/2022 | 35259932 | Saha, Gamm, Roy, Skala |
| Inhibition of B-cell lymphoma 2 family proteins alters optical redox ratio, mitochondrial polarization, and cell energetics independent of cell state | Journal of Biomedical Optics | Gillette et al. | 5/28/2022 | 35643815 | Saha, Gamm, Roy, Skala |
| Imaging the fetal nonhuman primate brain with SV2A positron emission tomography (PET) | European Journal of Nuclear Medicine and Molecular Imaging | Rossano et al. | 5/28/2022 | 35633376 | Tarantal, Hartigan-O'Connor, Segal |
| CRISPR Library Screening in Cultured Cardiomyocytes | Methods in Molecular Biology | DeLuca and Bursac | 5/27/2022 | 35618895 | Gerbach, Bursac, Truskey |
| Multivalent designed proteins neutralize SARS-CoV-2 variants of concern and confer protection against infection in mice | Science Translational Medicine | Hunt et al. | 5/25/2022 | 35412328 | Freedman |
| In situ autofluorescence lifetime assay of a photoreceptor stimulus response in mouse retina and human retinal organoids | Biomedical Optics Express | Samimi et al. | 5/18/2022 | 35781966 | Saha, Gamm, Roy, Skala |
| Structural biology of CRISPR-Cas immunity and genome editing enzymes | Nature Reviews Microbiology | Wang et al. | 5/13/2022 | 35562427 | Doudna, Banfield |
| The clinical progress of mRNA vaccines and immunotherapies | Nature Biotechnology | Barbier et al | 5/9/2022 | 35534554 | Gao, Anderson, Xue |
| Translational Utility of the Nonhuman Primate Model | Biological Psychiatry: Cognitive Neuroscience and Neuroimaging | Tarantal et al. | 5/6/2022 | 35283343 | Tarantal, Hartigan-O'Connor, Segal |
| Prioritization of autoimmune disease-associated genetic variants that perturb regulatory element activity in T cells | Nature Genetics | Mouri et al. | 5/5/2022 | 35513721 | Liu |
| Identification and Validation of CRISPR/Cas9 Off-Target Activity in Hematopoietic Stem and Progenitor Cells | Methods in Molecular Biology | Park et al. | 5/5/2022 | 35507169 | Gao, Anderson, Xue |
| Physiology assays in human kidney organoids | American Journal of Physiology - Renal Physiology | Freedman | 5/4/2022 | 35379001 | Freedman |
| CRISPR-Cas9 bends and twists DNA to read its sequence | Nature Structural & Molecular Biology | Cofsky et al. | 4/29/2022 | 35422516 | Doudna, Banfield |
| pH-Responsive Polymer Nanoparticles for Efficient Delivery of Cas9 Ribonucleoprotein With or Without Donor DNA | Advanced Materials | Xie et al. | 4/28/2022 | 35119139 | Gong, Emborg, Levine, Roy, Saha |
| Modelling ciliopathy phenotypes in human tissues derived from pluripotent stem cells with genetically ablated cilia | Nature Biomedical Engineering | Cruz et al. | 4/27/2022 | 35478224 | Freedman |
| Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome Editing | ACS Publications | Chen et al. | 4/21/2022 | 35446558 | Sontheimer, Khvorova, Watts, Wolfe |
| Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity | Human Gene Therapy | Tran et al. | 4/19/2022 | 35293222 | Gao, Anderson, Xue |
| Crystal structure of an RNA/DNA strand exchange junction | PLOS One | Cofsky et al. | 4/18/2022 | 35436289 | Doudna, Banfield |
| Derivation of extra-embryonic and intra-embryonic macrophage lineages from human pluripotent stem cells | Development | Bredemeyer et al. | 4/15/2022 | 35178561 | Hinson |
| An optimized FusX assembly-based technique to introduce mitochondrial TC-to-TT variations in human cell lines | STAR Protocols | Kar et al. | 4/15/2022 | 35496789 | Ekker, Clark |
| A High-Efficiency AAV for Endothelial Cell Transduction Throughout the Central Nervous System | Nature Cardiovascular Research | Krolak et al. | 4/13/2022 | 35571675 | Deverman |
| Myoblast deactivation within engineered human skeletal muscle creates a transcriptionally heterogeneous population of quiescent satellite-like cells | Biomaterials | Wang et al. | 4/7/2022 | 35633376 | Gersbach, Bursac, Truskey |
| Durability of transgene expression after rAAV gene therapy | Molecular Therapy | Muhuri et al. | 4/6/2022 | 35283274 | Gao, Anderson, Xue |
| Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells | PLOS One | Luks et al. | 4/6/2022 | 35385514 | Saltzman, Glazer |
| In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration | Nature Communications | Choi et al. | 4/5/2022 | 35383196 | Liu |
| CRISPR-free base editors with enhanced activity and expanded targeting scope in mitochondrial and nuclear DNA | Nature Biotechnology | Mok et al. | 4/4/2022 | 35379961 | Liu |
| A split prime editor with untethered reverse transcriptase and circular RNA template | Nature Biotechnology | Liu et al. | 4/4/2022 | 35379962 | Gao, Anderson, Xue | Sontheimer, Khvorova, Watts, Wolfe |
| Label-Free Imaging to Track Reprogramming of Human Somatic Cells | GEN Biotechnology | Molugu et al. | 4/1/2022 | 35586336 | Saha, Gamm, Roy, Skala |
| Robust, Durable Gene Activation In Vivo via mRNA-Encoded Activators | ACS Nano | Beyersdorf et al. | 3/31/2022 | 35357116 | Gersbach | Dahlman, Santangelo |
| Efficient targeted transgenesis of large donor DNA into multiple mouse genetic backgrounds using bacteriophage Bxb1 integrase | Scientific Reports | Low et al. | 3/31/2022 | 35361849 | Murray, Lutz |
| AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice | Nature | Wang et al. | 3/23/2022 | 35322228 | Gao, Anderson, Xue |
| Species-dependent in vivo mRNA delivery and cellular responses to nanoparticles | Nature Nanotechnology | Hatit et al. | 3/17/2022 | 35132167 | Dahlman, Santangelo |
| rAAV-delivered PTEN therapeutics for prostate cancer | Molecular Therapy - Nucleic Acids | Ai et al. | 3/8/2022 | 34976432 | Gao, Anderson, Xue |
| A self-immolative linker that releases thiols detects penicillin amidase and nitroreductase with high sensitivity via absorption spectroscopy | Chemical Communications | Espinoza et al. | 3/3/2022 | 35170593 | Bankiewicz, Murthy |
| Antispacer peptide nucleic acids for sequence-specific CRISPR-Cas9 modulation | Nucleic Acids Research | Economos et al. | 3/3/2022 | 35235944 | Glazer, Ly, Saltzman |
| Clinical magnetic hyperthermia requires integrated magnetic particle imaging | Wiley Interdisciplinary Reviews | Healy et al. | 3/3/2022 | 35238181 | Bulte |
| In Vivo Gene Editing in Lipid and Atherosclerosis Research | Methods in Molecular Biology | Giorgi et al. | 3/2/2022 | 35237996 | Heaney, Dickinson, Lagor |
| Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challenges | Science Translational Medicine | Schmidt et al. | 3/2/2022 | 35235338 | Gong, Emborg, Levine, Roy, Saha |
| Modeling injury and repair in kidney organoids reveals that homologous recombination governs tubular intrinsic repair | Science Translational Medicine | Gupta et al. | 3/2/2022 | 35235339 | Morizane, Lewis, Sabbisetti |
| Design of therapeutic biomaterials to control inflammation | Nature Reviews Materials | Tu et al. | 2/28/2022 | 35251702 | Leong |
| Treating Cardiovascular Disease with Liver Genome Engineering | Current Atherosclerosis Reports | Hurley and Lagor | 2/24/2022 | 35230602 | Heaney, Dickinson, Lagor |
| Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis | Proceedings of the National Academy of Sciences | Qiu et al. | 2/22/2022 | 35173043 | Chen, Liu, Xu |
| Nonhuman Primates in Translational Research | Annual Review of Animal Biosciences | Tarantal et al. | 2/15/2022 | 35167321 | Tarantal, Hartigan-O'Connor, Segal | Tarantal, Segal |
| AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies | Trends in Molecular Medicine | Yang et al. | 2/12/2022 | 33714697 | Gao, Anderson, Xue |
| A Human-derived Dual MRI/PET Reporter Gene System with High Translational Potential for Cell Tracking | Molecular Imaging and Biology | Shalaby et al. | 2/10/2022 | 35146614 | Ronald |
| AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset | Nature Neuroscience | Goertsen et al. | 1/25/2022 | 34887588 | Feng |
| Detecting Cardiovascular Protein-Protein Interactions by Proximity Proteomics | Circulation Research | Kushner et al. | 1/21/2022 | 35050691 | Hinson |
| Genome-wide detection of CRISPR editing in vivo using GUIDE-tag | Nature Communications | Liang et al. | 1/21/2022 | 35064134 | Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue |
| Rapid, accurate mapping of transgene integration in viable rhesus macaque embryos using enhanced specificity tagmentation-assisted PCR | Molecular Therapy Methods & Clinical Development | Ryu et al. | 1/19/2022 | 35211637 | Hennebold |
| Reading Frame Repair of TTN Truncation Variants Restores Titin Quantity and Functions | Circulation | Romano et al. | 1/18/2022 | 34905694 | Hinson |
| BRD2 inhibition blocks SARS-CoV-2 infection by reducing transcription of the host cell receptor ACE2 | Nature Cell Biology | Samelson et al. | 1/13/2022 | 35027731 | Conklin |
| Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins | Cell | Banskota et al. | 1/11/2022 | 35021064 | Liu | Chaikof |
| Gene editing in a Myo6 semi-dominant mouse model rescues auditory function | Molecular Therapy | Xue et al. | 1/5/2022 | 34174443 | Chen, Liu, Xu |
| A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver | Molecular Therapy | Zheng et al. | 1/5/2022 | 34998953 | Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue |
| Disruption of HIV-1 co-receptors CCR5 and CXCR4 in primary human T cells and hematopoietic stem and progenitor cells using base editing | Molecular Therapy | Knipping et al. | 1/5/2022 | 34737067 | Liu |
| Acid-Sensitive Surfactants Enhance the Delivery of Nucleic Acids | Molecular Pharmacology | Røise et al. | 12/21/2021 | 34931518 | Bankiewicz, Murthy |
| CRISPR/Cas9 ribonucleoprotein-mediated genome and epigenome editing in mammalian cells | Advanced Drug Delivery Reviews | Bloomer et al. | 12/20/2021 | 34942274 | Chen, Liu, Xu |
| Magnetic Iron Oxide Nanoparticles for Biomedical Applications | Current Opinion in Biomedical Engineering | Jiang et al. | 12/20/2021 | 35211662 | Bao, Lagor |
| The Coiled-Coil Forming Peptide (KVSALKE)5 Is a Cell Penetrating Peptide that Enhances the Intracellular Delivery of Proteins | Advanced Healthcare Materials | Li et al. | 12/16/2021 | 34861744 | Bankiewicz, Murthy |
| Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing | Nature Biotechnology | Anzalone et al. | 12/9/2021 | 34887556 | Liu |
| Light: A Magical Tool for Controlled Drug Delivery | Advanced Functional Materials | Tao et al. | 12/1/2021 | 34483808 | Leong |
| The FusX TALE Base Editor (FusXTBE) for Rapid Mitochondrial DNA Programming of Human Cells In Vitro and Zebrafish Disease Models In Vivo | The CRISPR Journal | Sabharwal et al. | 11/30/2021 | 34847747 | Ekker, Clark |
| CRISPR-enhanced human adipocyte browning as cell therapy for metabolic disease | Nature Communications | Tsagkaraki et al. | 11/26/2021 | 34836963 | Sontheimer, Khvorova, Watts, Wolfe |
| Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models | Nature Communications | Hakim et al. | 11/24/2021 | 34819506 | Gersbach, Bursac, Truskey | Gersbach | Asokan |
| Cross-validation of SARS-CoV-2 responses in kidney organoids and clinical populations | JCI Insight | Helms et al. | 11/12/2021 | 34767537 | Freedman |
| Gain-of-function cardiomyopathic mutations in RBM20 rewire splicing regulation and re-distribute ribonucleoprotein granules within processing bodies | Nature Communications | Fenix et al. | 11/3/2021 | 34732726 | Conklin |
| In vivo somatic cell base editing and prime editing | Molecular Therapy | Newby and Liu | 11/3/2021 | 34509669 | Liu |
| Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy | Molecular Therapy | Pickar-Oliver et al. | 11/3/2021 | 34509668 | Gersbach, Bursac, Truskey | Asokan |
| Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome Editing | ACS Publications | Qiu et al. | 11/2/2021 | 34668716 | Chen, Liu, Xu |
| Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo | Nature Communications | Ibraheim et al. | 11/1/2021 | 34725353 | Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue |
| Enhanced prime editing systems by manipulating cellular determinants of editing outcomes | Cell | Chen et al. | 10/28/2021 | 34653350 | Liu |
| Neuromuscular Development and Disease: Learning From in vitro and in vivo Models | Frontiers in Cell and Developmental Biology | Fralish et al. | 10/27/2021 | 34778273 | Gersbach, Bursac, Truskey | Asokan |
| AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway | Molecular Therapy | Liang et al. | 10/23/2021 | 34695545 | Gao, Anderson, Xue | Heaney, Dickinson, Lagor |
| Electrophoretic mobility shift as a molecular beacon-based readout for miRNA detection | Biosensors and Bioelectronics | Oliveira Jr et al. | 10/21/2021 | 34062334 | Ghiran |
| 5' modifications improve potency and efficacy of DNA donors for precision genome editing | eLife | Ghanta et al. | 10/19/2021 | 34665130 | Sontheimer, Khvorova, Watts, Wolfe |
| SnRNA sequencing defines signaling by RBC-derived extracellular vesicles in the murine heart | Life Science Alliance | Valkov et al. | 10/18/2021 | 34663679 | Ghiran |
| Deletion and replacement of long genomic sequences using prime editing | Nature Biotechnology | Jiang et al. | 10/14/2021 | 34650270 | Gao, Anderson, Xue |
| Disulfide-compatible phage-assisted continuous evolution in the periplasmic space | Nature Communications | Morrison et al. | 10/13/2021 | 34645844 | Liu |
| Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors | Nucleic Acids Research | Krishnamurthy et al. | 10/11/2021 | 34520545 | McCray | Liu |
| Kidney development to kidney organoids and back again | Seminars in Cell & Developmental Biology | Gupta and Morizane | 10/6/2021 | 34627669 | Morizane, Lewis, Sabbisetti |
| Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs | Nature Biomedical Engineering | Lokugamage et al. | 10/6/2021 | 34616046 | Dahlman, Santangelo |
| Engineered pegRNAs improve prime editing efficiency | Nature Biotechnology | Nelson et al. | 10/4/2021 | 34608327 | Liu |
| In Vivo MRI Tracking of Tumor Vaccination and Antigen Presentation by Dendritic Cells | Molecular Imaging and Biology | Bulte et al. | 9/28/2021 | 34581954 | Bulte |
| Synthetic Biology Approaches for Engineering Next-Generation Adenoviral Gene Therapies | ACS Publications | Collins and Curiel | 9/28/2021 | 34415739 | Curiel |
| External stimuli-responsive nanoparticles for spatially and temporally controlled delivery of CRISPR-Cas genome editors | Biomaterials Science | Xie et al. | 9/14/2021 | 34286726 | Gong, Emborg, Levine, Roy, Saha |
| Receptor Switching in Newly Evolved Adeno-associated Viruses | Journal of Virology | Havlik et al. | 9/9/2021 | 34232726 | Asokan |
| Cortical overgrowth in a preclinical forebrain organoid model of CNTNAP2-associated autism spectrum disorder | Nature Communications | de Jong et al. | 9/1/2021 | 34471112 | Leong |
| Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3 | Proceeding of the National Academy of Sciences in the United States of America | Qiu et al. | 9/1/2021 | 33649229 | Chen, Liu, Xu |
| DNA interference states of the hypercompact CRISPR-CasΦ effector | Nature Structural & Molecular Biology | Pausch et al. | 8/28/2021 | 34381246 | Doudna, Banfield |
| Easy-Prime: a machine learning-based prime editor design tool | Genome Biology | Li et al. | 8/19/2021 | 34412673 | Tsai |
| Transgenic mice for in vivo epigenome editing with CRISPR-based systems | Nature Methods | Gemberling et al. | 8/18/2021 | 34341582 | Gersbach | Asokan |
| Allele-Specific Gene Editing Rescues Pathology in a Human Model of Charcot-Marie-Tooth Disease Type 2E | Frontiers in Cell and Developmental Biology | Feliciano et al. | 8/16/2021 | 35633376 | Conklin |
| In vivo targeted delivery of nucleic acids and CRISPR genome editors enabled by GSH-responsive silica nanoparticles | Journal of Controlled Release | Wang et al. | 8/10/2021 | 34174352 | Gong, Emborg, Levine, Roy, Saha |
| Actinin BioID reveals sarcomere crosstalk with oxidative metabolism through interactions with IGF2BP2 | Cell Reports | Ladha et al. | 8/10/2021 | 34380038 | Hinson |
| Vector Strategies to Actualize B Cell-Based Gene Therapies | Journal of Immunology | Jeske et al. | 8/1/2021 | 34321286 | Curiel |
| Label-free imaging for quality control of cardiomyocyte differentiation | Nature Communications | Qian et al. | 7/28/2021 | 34321477 | Saha, Gamm, Roy, Skala |
| Recent innovations in fluorescence lifetime imaging microscopy for biology and medicine | Journal of Biomedical Optics | Datta et al. | 7/26/2021 | 34247457 | Saha, Gamm, Roy, Skala |
| Mechanisms of angiogenic incompetence in Hutchinson-Gilford progeria via downregulation of endothelial NOS | Aging Cell | Gete et al. | 7/20/2021 | 34086398 | Liu |
| Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science | Annual Review of Biomedical Engineering | Abdeen et al. | 7/13/2021 | 33909475 | Gersbach | Gersbach, Bursac, Truskey |
| Escaping the endosome: assessing cellular trafficking mechanisms of non-viral vehicles | Journal of Controlled Release | Xu et al. | 7/10/2021 | 34077782 | Saltzman, Glazer |
| rAAV-based and intraprostatically delivered miR-34a therapeutics for efficient inhibition of prostate cancer progression | Gene Therapy | Ai et al. | 7/6/2021 | 34226687 | Gao, Anderson, Xue |
| Diverse ATPase Proteins in Mobilomes Constitute a Large Potential Sink for Prokaryotic Host ATP | Frontiers in Microbiology | Shim et al. | 7/7/21 | 34305853 | Doudna, Banfield |
| Targeting the De Novo Purine Synthesis Pathway Through Adenylosuccinate Lyase Depletion Impairs Liver Cancer Growth by Perturbing Mitochondrial Function | Hepatology | Jiang et al. | 7/1/2021 | 33336367 | Gao, Anderson, Xue |
| Base editing of haematopoietic stem cells rescues sickle cell disease in mice | Nature | Newby et al. | 7/1/2021 | 34079130 | Liu | Tsai |
| Mucopolysaccharidoses type I gene therapy | Journal of Inherited Metabolic Disease | Hurt et al. | 6/29/2021 | 34189746 | Curiel |
| Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning | Nature Biotechnology | Koblan et al. | 6/28/2021 | 34183861 | Liu | Chaikof |
| 3D cell culture models: Drug pharmacokinetics, safety assessment, and regulatory consideration | Clinical and Translational Science | Wang et al. | 6/16/2021 | 33982436 | Freedman |
| Induced pluripotent stem cells provide mega insights into kidney disease | Kidney International | Luciani et al. | 6/15/2021 | 32571490 | Freedman |
| Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy | Molecular Therapy Methods & Clinical Development | Marquez Loza et al. | 6/11/2021 | 33768133 | McCray |
| Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repair | Cell | Wilde et al. | 6/10/2021 | 34043941 | Feng |
| SARS-CoV-2 infection induces the activation of tissue factor-mediated coagulation by activation of acid sphingomyelinase | Blood | Wang et al. | 6/1/2021 | 34075401 | Yi |
| In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness | Science Translational Medicine | Yeh et al. | 6/1/2021 | 32493795 | Liu | Chen, Liu, Xu |
| Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering | Cell Reports | Hamilton et al. | 6/1/2021 | 34077734 | Doudna, Banfield |
| Generation and Genetic Correction of USH2A c.2299delG Mutation in Patient-Derived Induced Pluripotent Stem Cells | Genes (Basel) | Liu et al. | 5/25/2021 | 34070435 | Chen, Liu, Xu |
| Quantification of Cellular Densities and Antigenic Properties using Magnetic Levitation | Journal of Visualized Experiements | Thompson et al. | 5/17/2021 | 34057455 | Ghiran |
| Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens? | Frontiers in Immunology | Zhan et al. | 5/11/2021 | 34046041 | Gao, Anderson, Xue |
| Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease | Communications Biology | Wang et al. | 5/5/2021 | 33953320 | Gersbach, Bursac, Truskey |
| Sarcomere function activates a p53-dependent DNA damage response that promotes polyploidization and limits in vivo cell engraftment | Cell Reports | Pettinato et al. | 5/4/2021 | 33951429 | Hinson |
| Serum Concentrations of Per- and Polyfluoroalkyl Substances and Risk of Renal Cell Carcinoma | Journal of the National Cancer Institute | Shearer et al. | 5/4/2021 | 32944748 | Morizane, Lewis, Sabbisetti |
| A Versatile and Robust Platform for the Scalable Manufacture of Biomimetic Nanovaccines | Advanced Science (Weinh) | Hu et al. | 5/1/2021 | 34386315 | Leong |
| Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction | Frontiers in Immunology | Muhri et al. | 4/28/2021 | 33995418 | Gao, Anderson, Xue |
| Targeting the Apoa1 locus for liver-directed gene therapy | Molecular Therapy Methods & Clinical Development | De Giorgi et al. | 4/24/2021 | 34141821 | Bao, Lagor | Heaney, Dickinson, Lagor |
| Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice | Nature Communications | Liu et al. | 4/9/2021 | 33837189 | Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue |
| The NIH Somatic Cell Genome Editing program | Nature | Saha, K., Sontheimer, E.J., Brooks, P.J. et al. | 4/7/2021 | 33828315 | SCGE Consortium |
| A CRISPR-Cas9-integrase complex generates precise DNA fragments for genome integration | Nucleic Acids Research | Jakhanwal et al. | 4/6/2021 | 33693715 | Doudna, Banfield |
| VectorMOD: Method for Bottom-Up Proteomic Characterization of rAAV Capsid Post-Translational Modifications and Vector Impurities | Frontiers in Immunology | Rumachik et al. | 4/1/2021 | 33868302 | McDevitt |
| Large-scale molecular epidemiological analysis of AAV in a cancer patient population | Oncogene | Qin et al. | 3/29/2021 | 33782545 | Gao, Anderson, Xue |
| PEGylation of poly(amine-co-ester) polyplexes for tunable gene delivery | Biomaterials | Grun et al | 3/24/2021 | 33813260 | Saltzman, Glazer |
| Loss of Sarcomeric Proteins via Upregulation of JAK/STAT Signaling Underlies Interferon-γ-induced Contractile Deficit in Engineered Human Myocardium | Acta Biomaterialia | Zhan et al. | 3/8/2021 | 35633376 | Gersbach, Bursac, Truskey |
| Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression | Genome Biology | Gao et al. | 3/16/2021 | 33722289 | Tsai | Liu |
| SARS-CoV-2 infection of human iPSC-derived cardiac cells reflects cytopathic features in hearts of patients with COVID-19 | Science Translational Medicine | Perez-Bermejo et al. | 3/15/2021 | 33723017 | McDevitt |
| Nanoparticles for delivery of agents to fetal lungs | Acta Biomaterialia | Ullrich et al. | 3/15/2021 | 33484911 | Saltzman, Glazer |
| Therapeutic RNA Delivery for COVID and Other Diseases | Advanced Healthcare Materials | Dobrowolski et al. | 3/4/2021 | 33661555 | Dahlman, Santangelo |
| Magnetic Forces Enable Control of Biological Processes In Vivo | Journal of Applied Mechanics | Bao | 3/1/2021 | 34168385 | Bao, Lagor |
| Tissue-Engineered Skeletal Muscle Models to Study Muscle Function, Plasticity, and Disease. | Frontiers in Physiology | Khodabukus | 2/26/2021 | 33716768 | Gersbach, Bursac, Truskey |
| COVID-19 CG enables SARS-CoV-2 mutation and lineage tracking by locations and dates of interest | eLife | Chen et al. | 2/23/2021 | 33620031 | Deverman |
| Massively parallel assessment of human variants with base editor screens | Cell | Hanna et al. | 2/18/2021 | 33606977 | Liu |
| Precision genome editing using cytosine and adenine base editors in mammalian cells | Nature Protocols | Huang et al. | 2/16/2021 | 33462442 | Liu |
| PrimeDesign software for rapid and simplified design of prime editing guide RNAs | Nature Communications | Hsu et al. | 2/15/2021 | 33589617 | Liu |
| Soluble CD40L activates soluble and cell-surface integrin αvβ3, α5β1, and α4β1 by binding to the allosteric ligand-binding site (site 2) | Journal of Biological Chemistry | Takada et al. | 2/9/2021 | 33571526 | Lam, Cheng |
| Viral vector platforms within the gene therapy landscape. | Signal Transduction and Targeted Therapy | Bulcha et al. | 2/8/2021 | 33558455 | Gao, Anderson, Xue |
| Gene-based therapies for neurodegenerative diseases | Nature Neuroscience | Sun et al. | 2/1/2021 | 33526943 | Gong, Emborg, Levine, Roy, Saha |
| Gene Regulatory Network Analysis and Engineering Directs Development and Vascularization of Multilineage Human Liver Organoids | Cell Systems | Velazquez et al. | 1/20/2021 | 332907415 | Kiani |
| Protein-based nanoplatform for detection of tumorigenic polyps in the colon via noninvasive mucosal routes | Pharmaceutical Patent Analysis | Chen et al. | 1/20/2021 | 33467938 | Lam, Cheng |
| Engineering highly efficient backsplicing and translation of synthetic circRNAs | Molecular Therapy - Nucleic Acids Journal | Meganck et al. | 1/16/2021 | 336142328 | Asokan |
| Tools for experimental and computational analyses of off-target editing by programmable nucleases | Nature Protocols | Bao et al. | 1/16/2021 | 33288953 | Bao, Lagor |
| CRISPR/Cas9 gene editing for curing sickle cell disease | Transfusion and Apheresis Science | Park and Bao | 1/10/2021 | 33455878 | Bao, Lagor |
| Exercise mimetics and JAK inhibition attenuate IFN-γ-induced wasting in engineered human skeletal muscle | Science Advances | Chen et al. | 1/7/2021 | 33523949 | Gersbach, Bursac, Truskey |
| In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice. | Nature | Kloban et al. | 1/6/2021 | 33408413 | Liu |
| Overcoming innate immune barriers that impede AAV gene therapy vectors | The Journal of Clinical Investigation | Muhuri et al. | 1/4/2021 | 33393506 | Gao, Anderson, Xue |
| Frame-Hydrogel Methodology for Engineering Highly Functional Cardiac Tissue Constructs | Methods in Molecular Biology | Heifer et al. | 1/1/2021 | 32857373 | Gersbach, Bursac, Truskey |
| Targeting de novo purine synthesis pathway via ADSL depletion impairs liver cancer growth by perturbing mitochondrial function | Hepatology | Jiang et al. | 12/17/2020 | 33336367 | Gao, Anderson, Xue |
| Multicellular Systems to Translate Somatic Cell Genome Editors to Humans | Current Opinion in Biotechnology | Hernandez-Gordillo et al. | 12/16/2020 | 33718690 | Kiani |
| The dawn of non-human primate models for neurodevelopmental disorders. | Current Opinion in Genetics and Development | Aida et al. | 12/1/2020 | 33718690 | Feng |
| Advances in microphysiological blood-brain barrier (BBB) models towards drug delivery | Current Opinion in Biotechnology | Lee et al. | 12/1/2020 | 32711361 | Leong |
| Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation Screens | Cell Reports | Black et al. | 12/1/2020 | 33264623 | Gersbach |
| Poison Exon Splicing Regulates a Coordinated Network of SR Protein Expression during Differentiation and Tumorigenesis. | Molecular Cell | Leclair et al. | 11/19/2020 | 33176162 | Hinson |
| Phage-assisted continuous and non-continuous evolution | Nature Protocols | Miller et al. | 11/16/2020 | 33199872 | Liu |
| The once and future gene therapy | Nature Communications | Bulaklak et al. | 11/16/2020 | 33199717 | Gersbach, Bursac, Truskey | Gersbach |
| Delayed urea differential enhancement CEST (dudeCEST)-MRI with T 1 correction for monitoring renal urea handling | Magnetic Resonance in Medicine | Shin et al. | 11/12/2020 | 33180343 | Vandsburger |
| Adenoviral Vectors for In Vivo Delivery of CRISPR-Cas Gene Editors | Journal of Controlled Release | Boucher et al. | 11/10/2020 | 32891680 | Curiel |
| Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical Applications | Mater Today (Kidlington) | Hu et al. | 11/2/2020 | 34421329 | Leong |
| Advances in Alpha-1 Antitrypsin Gene Therapy | America Journal of Respiratory Cell and Nolecular Biology | Lorincz et al. | 11/1/2020 | 32668173 | Curiel |
| The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction. | Journal of Virology | Madigan et al. | 10/14/2020 | 32817219 | Asokan |
| A Versatile Nonviral Delivery System for Multiplex Gene-Editing in the Liver | Advanced Materials | Gong et al. | 10/14/2020 | 33053221 | Leong |
| 3D kidney organoids for bench-to-bedside translation | Journal of Molecular Medicine | Gupta et al. | 10/9/2020 | 33034708 | Morizane, Lewis, Sabbisetti |
| Development of a Cardiac Sarcomere Functional Genomics Platform to Enable Scalable Interrogation of Human TNNT2 Variants | Circulation | Pettinato et al. | 10/7/2020 | 33025817 | Hinson |
| Opportunities and limitations of genetically modified nonhuman primate models for neuroscience research | Proceeding of the National Academy of Sciences in the United States of America | Feng et al. | 9/29/2020 | 32817435 | Feng |
| COVID-19 CG: Tracking SARS-CoV-2 mutations by locations and dates of interest | bioRxiv | Chen et al. | 9/28/2020 | 32995794 | Deverman |
| In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy | Molecular Therapy Methods & Clinical Development | Kwon et al. | 9/28/2020 | 33145368 | Asokan | Gersbach, Bursac, Truskey |
| Synthetic immunomodulation with a CRISPR super-repressor in vivo | Nature Cell Biology | Moghadam et al. | 9/22/2020 | 32884147 | Kiani |
| Treating Cystic Fibrosis with mRNA and CRISPR | Human Gene Therapy | Da Silva Sanchez et al. | 9/16/2020 | 32799680 | Dahlman, Santangelo |
| Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach | Journal of Virology | Havalik et al. | 9/15/2020 | 32669336 | Asokan |
| SARS-CoV-2 infection of human iPSC-derived cardiac cells predicts novel cytopathic features in hearts of COVID-19 patients | bioRxiv | Pérez-Bermejo et al. | 9/12/2020 | 32884147 | McDevitt |
| Direct Functional Protein Delivery with a Peptide into Neonatal and Adult Mammalian Inner Ear In Vivo | Molecular Therapy Methods & Clinical Development | Zhang et al. | 9/11/2020 | 32953927 | Chen, Liu, Xu |
| Rapid, precise quantification of large DNA excisions and inversions by ddPCR. | Scientific Reports | Watry et al. | 9/10/2020 | 32913194 | McDevitt |
| New Directions in Pulmonary Gene Therapy | Human Gene Therapy | Vu et al. | 9/3/2020 | 32814451 | McCray |
| Imidazole-Based Synthetic Lipidoids for In Vivo mRNA Delivery into Primary T Lymphocytes | Angewandte Chemie International Edition | Zhao et al. | 8/28/2020 | 32662132 | Chen, Liu, Xu |
| mRNA Delivery Using Bioreducible Lipidoid Nanoparticles Facilitates Neural Differentiation of Human Mesenchymal Stem Cells | Advanced Healthcare Materials | Zhao et al. | 8/20/2020 | 32815325 | Chen, Liu, Xu |
| A pH-responsive silica-metal-organic framework hybrid nanoparticle for the delivery of hydrophilic drugs, nucleic acids, and CRISPR-Cas9 genome-editing machineries. | Journal of Controlled Release | Wang et al. | 8/10/2020 | 32380204 | Gong, Emborg, Levine, Roy, Saha |
| A scoutRNA Is Required for Some Type V CRISPR-Cas Systems | Molecular Cell | Harrington et al. | 8/6/2020 | 32645367 | Doudna, Banfield |
| NDAT Targets PI3K-Mediated PD-L1 Upregulation to Reduce Proliferation in Gefitinib-Resistant Colorectal Cancer. | Cells | Huang et al. | 8/3/2020 | 32756527 | Lam, Cheng |
| Finding order in slow axonal transport | Current Opinion in Neurobiology | Wang et al. | 8/1/2020 | 32361600 | Gong, Emborg, Levine, Roy, Saha |
| DNA capture by a CRISPR-Cas9-guided adenine base editor. | Science | Al-Shayeb et al. | 7/31/2020 | 32732424 | Doudna, Banfield | Liu |
| Epigenetic transcriptional reprogramming by WT1 mediates a repair response during podocyte injury. | Science Advances | Ettou et al. | 7/24/2020 | 32754639 | Morizane, Lewis, Sabbisetti |
| Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning. | Cell | Arbab et al. | 7/23/2020 | 32533916 | Liu |
| CRISPR-CasΦ from huge phages is a hypercompact genome editor. | Science | Pausch et al. | 7/17/2020 | 32675376 | Doudna, Banfield |
| Combinatorial Library of Cyclic Benzylidene Acetal-Containing pH-Responsive Lipidoid Nanoparticles for Intracellular mRNA Delivery. | Bioconjugate Chemistry | Li et al. | 7/15/2020 | 32520527 | Chen, Liu, Xu |
| Tissue-Engineered Human Myobundle System as a Platform for Evaluation of Skeletal Muscle Injury Biomarkers. | Toxicological Sciences | Khodabukus et al. | 7/1/2020 | 32294208 | Gersbach, Bursac, Truskey |
| Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties | Nature Communications | Hsu et al. | 6/30/2020 | 32606306 | Gao, Anderson, Xue |
| The delivery challenge: fulfilling the promise of therapeutic genome editing | Nature Biotechnology | van Haasteren et al. | 6/29/2020 | 32601435 | Bankiewicz, Murthy |
| Programmable m6A modification of cellular RNAs with a Cas13-directed methyltransferase. | Nature Biotechnology | Wilson et al. | 6/29/2020 | 32601430 | Liu |
| Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection | Science Advances | Ma et al. | 6/24/2020 | 32832671 | Chen, Liu, Xu |
| Polymeric vehicles for nucleic acid delivery. | Advanced Drug Delivery Reviews | Piotrowski-Daspit et al. | 6/23/2020 | 32585159 | Saltzman, Glazer |
| Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors. | Nature Biotechnology | Anzalone et al. | 6/22/2020 | 32572269 | Liu |
| A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing. | Nature | Mok et al. | 6/22/2020 | 32641830 | Liu |
| Single C-to-T substitution using engineered APOBEC3G-nCas9 base editors with minimum genome- and transcriptome-wide off-target effects | Science Advances | Lee et al. | 6/15/2020 | 32832622 | Bao, Lagor |
| CHANGE-seq reveals genetic and epigenetic effects on CRISPR-Cas9 genome-wide activity. | Nature Biotechnology | Lazzarotto et al. | 6/15/2020 | 32541958 | Tsai |
| Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells. | Cell Stem Cell | Nagpal et al. | 6/4/2020 | 32320679 | Sontheimer, Khvorova, Watts, Wolfe |
| AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9. | Molecular Therapy | Li et al. | 6/3/2020 | 32348718 | Bao, Lagor | Heaney, Dickinson, Lagor |
| Redirecting Vesicular Transport to Improve Non-viral Delivery of Molecular Cargo | Advanced Biology | Mao et al. | 6/21/2020 | 33179869 | Gersbach, Bursac, Truskey |
| Can we mimic skeletal muscles for novel drug discovery? | Expert Opinion on Drug Delivery | Broer et al. | 6/1/2020 | 32122176 | Gersbach, Bursac, Truskey |
| Non-viral nanoparticles for CRISPR-based genome editing: is it just a simple adaption of what have been developed for nucleic acid delivery? | Biomacromolecules | Qiu et al. | 5/31/2020 | 31342740 | Chen, Liu, Xu |
| Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligands | Bioengineering & Translational Medicine | Gan et al. | 5/27/2020 | 33758781 | Dahlman, Santangelo |
| YAP1 withdrawal in hepatoblastoma drives therapeutic differentiation of tumor cells to functional hepatocyte-like cells. | Hepatology | Smith et al. | 5/26/2020 | 32452550 | Gao, Anderson, Xue | Liu |
| The developing toolkit of continuous directed evolution | Nature Chemical Biology | Morrison et al. | 5/22/2020 | 32444838 | Liu |
| Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors | Molecular Therapy, Methods & Clinical Development | Rumachik et al. | 5/22/2020 | 32995354 | McDevitt |
| Protein and mRNA Delivery Enabled by Cholesteryl-Based Biodegradable Lipidoid Nanoparticles. | Angewandte Chemie International Edition in English | Li et al. | 5/21/2020 | 32438474 | Chen, Liu, Xu |
| Effect of luminal flow on doming of mpkCCD cells in a 3D perfusable kidney cortical collecting duct model | American Journal of Physiology-Cell Physiology | Rein et al. | 5/13/2020 | 32401606 | Morizane, Lewis, Sabbisetti |
| Transcriptomic and Proteostasis Networks of CFTR and the Development of Small Molecule Modulators for the Treatment of Cystic Fibrosis Lung Disease | Genes (Basel) | Strub and McCray | 5/13/2020 | 32414011 | McCray |
| Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators | Stem Cell Reports | Kwon et al. | 5/12/2020 | 32330446 | Gersbach, Bursac, Truskey | Asokan | Gersbach |
| Advances in genome editing for genetic hearing loss. | Advanced Drug Delivery Reviews | Ding et al. | 5/7/2020 | 32387678 | Bao, Lagor |
| A New Gorilla Adenoviral Vector with Natural Lung Tropism Avoids Liver Toxicity and Is Amenable to Capsid Engineering and Vector Retargeting | Journal of Virology | Lu et al. | 5/4/2020 | 32102889 | Curiel |
| Once upon a dish: engineering multicellular systems | Development | Haase et al. | 5/4/2020 | 32366376 | Freedman |
| Positron emission tomography imaging of novel AAV capsids maps rapid brain accumulation | Nature Communications | Seo et al. | 4/30/2020 | 32355221 | Lam, Cheng |
| Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope. | Nature Communications | Jiang et al. | 4/24/2020 | 32332735 | Gao, Anderson, Xue | Liu |
| Prime genome editing in rice and wheat | Nature Biotechnology | Lin et al. | 3/16/2020 | 32393904 | Liu |
| Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity. | Nature Biotechnology | Richter et al. | 3/16/2020 | 32433547 | Liu |
| Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases | Theranostics | Wu et al. | 3/15/2020 | 32292501 | Gao, Anderson, Xue |
| Targeted Delivery of Secretory Promelittin via Novel Poly(lactone‐co‐β‐amino ester) Nanoparticles for Treatment of Breast Cancer Brain Metastases | Advanced Science (Weinh) | Zhou et al. | 3/7/2020 | 32154067 | Zhou |
| Profiling APOL1 Nephropathy Risk Variants in Genome-Edited Kidney Organoids with Single-Cell Transcriptomics. | Kidney360 | Liu et al. | 3/1/2020 | 32656538 | Freedman |
| Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications | Biomaterials | Li et al. | 3/1/2020 | 31945616 | Chen, Liu, Xu |
| Adenine base editing in an adult mouse model of tyrosinaemia. | Nature Biomedical Engineering | Song et al. | 2/25/2020 | 31740768 | Liu | Gao, Anderson, Xue |
| Poly(Lactic-co-Glycolic Acid) Nanoparticle Delivery of Peptide Nucleic Acids In Vivo. | Methods in Molecular Biology | Oyaghire et al. | 2/23/2020 | 32088877 | Saltzman, Glazer |
| High-throughput quantitative microscopy-based half-life measurements of intravenously injected agents | Proceeding of the National Academy of Sciences in the United States of America | Bracaglia et al. | 2/18/2020 | 32015123 | Saltzman, Glazer |
| Site-Specific Post-translational Surface Modification of Adeno-Associated Virus Vectors Using Leucine Zippers | ACS Synthetic Biology | Thadani et al. | 2/18/2020 | 32068391 | Bao, Lagor |
| Clades of huge phages from across Earth's ecosystems | Nature | Al-Shayeb et al. | 2/12/2020 | 32051592 | Doudna, Banfield |
| The promise and challenge of therapeutic genome editing | Nature | Doudna | 2/12/2020 | 32051598 | Doudna, Banfield |
| Quantitating Endosomal Escape of a Library of Polymers for mRNA Delivery | Nano Letters | Jiang et al. | 2/12/2020 | 32003222 | Saltzman, Glazer |
| Continuous evolution of SpCas9 variants compatible with non-G PAMs. | Nature Biotechnology | Miller et al. | 2/10/2020 | 32042170 | Liu |
| Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors. | Nature Biotechnology | Doman et al. | 2/10/2020 | 32042165 | Liu |
| Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair. | Molecules | Economos et al. | 2/8/2020 | 32046275 | Saltzman, Glazer | Glazer, Ly, Saltzman |
| Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs. | The Journal of Investigative Dermatology | Osborn et al. | 2/1/2020 | 31437443 | Liu |
| From GWAS Association to Function: Candidate Gene Screening Within Insulin Resistance-Associated Genomic Loci Using a Preadipocyte Differentiation Model | Circulation Research | Ladha et al. | 1/31/2020 | 31999535 | Hinson |
| Detection of Extracellular Vesicle RNA Using Molecular Beacons. | iScience | Oliveira Jr, et al. | 1/24/2020 | 31958756 | Ghiran |
| Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses. | Nature Biomedical Engineering | Levy et al. | 1/4/2020 | 31937940 | Liu | Chen, Liu, Xu |
| Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings. | Human Gene Therapy Methods | Maynard et al. | 12/19/2019 | 31855083 | McDevitt |
| Structures of Neisseria meningitidis Cas9 Complexes in Catalytically Poised and Anti-CRISPR-Inhibited States. | Molecular Cell | Sun et al. | 12/19/2019 | 31668930 | Sontheimer, Khvorova, Watts, Wolfe |
| Optimizing biodegradable nanoparticle size for tissue-specific delivery. | Journal of Controlled Release | Mandl et al. | 11/28/2019 | 31654688 | Saltzman, Glazer |
| Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids | PLOS ONE | Huang et al. | 11/14/2019 | 31725765 | Deverman |
| Genome editing of HBG1 and HBG2 to induce fetal hemoglobin | Blood Advances | Métais et al. | 11/12/2019 | 31698466 | Tsai |
| Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia | Nature Communications | Sateesh et al. | 10/28/2019 | 31659165 | McCray |
| Search-and-replace genome editing without double-strand breaks or donor DNA | Nature | Anzalone et al. | 10/21/2019 | 31634902 | Liu |
| All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo. | Genome Biology | Ibraheim et al. | 9/19/2019 | 30231914 | Gao, Anderson, Xue |
| A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing | Nature Nanotechnology | Chen et al. | 9/9/2019 | 31501532 | Gong, Emborg, Levine, Roy, Saha |
| Nonviral Nanoparticles for CRISPR-Based Genome Editing: Is It Just a Simple Adaption of What Have Been Developed for Nucleic Acid Delivery? | Biomacromolecules | Qiu et al. | 9/9/2019 | 31342740 | Chen, Liu, Xu |
| Non-invasive Imaging of Renal Urea Handling by Chemical Exchange Saturation Transfer (CEST)-MRI | Magnetic Resonance in Medicine | Shin et al. | 9/4/2019 | 35633376 | Vandsburger |
| High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs. | Human Gene Therapy | Xu et al. | 8/30/2019 | 31072208 | Gao, Anderson, Xue |
| Tissue-restricted genome editing in vivo specified by microRNA-repressible anti-CRISPR proteins | RNA | Lee et al. | 8/22/2019 | 31439808 | Gao, Anderson, Xue | Sontheimer, Khvorova, Watts, Wolfe |
| Mapping cis-regulatory chromatin contacts in neural cells links neuropsychiatric disorder risk variants to target genes. | Nature Genetics | Song et al. | 7/31/2019 | 31367015 | McDevitt |
| Continuous evolution of base editors with expanded target compatibility and improved activity | Nature Biotechnology | Thuronyi et al. | 7/22/2019 | 31332326 | Liu |
| Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis. | Nature Communications | Yang et al. | 7/4/2019 | 31273195 | Gao, Anderson, Xue |
| An anionic human protein mediates cationic liposome delivery of genome editing proteins into mammalian cells | Nature Communications | Kim et al. | 7/2/2019 | 31266953 | Liu |
| Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles. | Advanced Materials | Liu et al. | 6/19/2019 | 31215123 | Chen, Liu, Xu |
| Depletion of TRRAP Induces p53-Independent Senescence in Liver Cancer by Down-Regulating Mitotic Genes. | Hepatology | Kwan et al. | 6/12/2019 | 31188495 | Gao, Anderson, Xue |
| Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors. | Nature Biotechnology | Huang et al. | 6/10/2019 | 31110355 | Liu |
| Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity | JCI Insight | Xiao et al. | 5/21/2019 | 31112525 | Gao, Anderson, Xue |
| Adeno-associated virus vector as a platform for gene therapy delivery. | Nature Reviews Drug Discovery | Wang et al. | 5/18/2019 | 30710128 | Gao, Anderson, Xue |
| Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks. | Nature Communications | Rees et al. | 5/17/2019 | 31101808 | Liu |
| Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy | Human Gene Therapy | Gong et al. | 5/15/2019 | 30358470 | Gao, Anderson, Xue |
| Analysis and minimization of cellular RNA editing by DNA adenine base editors | Science Advances | Rees et al. | 5/8/2019 | 31086823 | Liu |
| Nucleic Acid Therapeutics for Neurological Diseases | Neurotherapeutics | Watts et al. | 4/29/2019 | 31037650 | Sontheimer, Khvorova, Watts, Wolfe |
| Unbiased detection of CRISPR off-targets in vivo using DISCOVER-Seq | Science | Wienert et al. | 4/19/2019 | 31000663 | McDevitt |
| CRISPR-SONIC: targeted somatic oncogene knock-in enables rapid in vivo cancer modeling. | Genome Medicine | Mou et al. | 4/16/2019 | 30987660 | Gao, Anderson, Xue |
| An orange calcium-modulated bioluminescent indicator for non-invasive activity imaging. | Nature Chemical Biology | Oh et al. | 4/1/2019 | 30936501 | McDevitt |
| Kidney Organoids in Translational Medicine: Disease Modeling and Regenerative Medicine | Developmental Dynamics | Miyoshi et al. | 3/26/2019 | 35633376 | Morizane, Lewis, Sabbisetti |
| Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression. | Immunity | Martinez-Navio et al. | 3/19/2019 | 30850342 | Gao, Anderson, Xue |
| Anti-drug Antibody Responses Impair Prophylaxis Mediated by AAV-Delivered HIV-1 Broadly Neutralizing Antibodies | Molecular Therapy | Gardner et al. | 3/6/2019 | 30704961 | Gao, Anderson, Xue |
| A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing | Molecular Cell | Edraki et al. | 2/21/2019 | 30581144 | Gao, Anderson, Xue |
| Enhancing the In Vitro and In Vivo Stabilities of Polymeric Nucleic Acid Delivery Nanosystems. | Bioconjugate Chemistry | Wang et al. | 2/20/2019 | 30592619 | Gong, Emborg, Levine, Roy, Saha |
| Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System | Methods in Molecular Biology | Gessler et al. | 2/20/2019 | 30783972 | Gao, Anderson, Xue |
| Quantitative and Digital Droplet-Based AAV Genome Titration | Methods in Molecular Biology | Sanmiguel et al. | 2/20/2019 | 30783968 | Gao, Anderson, Xue |
| Simultaneous targeting of linked loci in mouse embryos using base editing | Scientific Reports | Lee et al. | 2/7/2019 | 30733567 | Liu |
| Integrating Combinatorial Lipid Nanoparticle and Chemically Modified Protein for Intracellular Delivery and Genome Editing | Accounts of Chemical Research | Chang et al. | 12/26/2018 | 30586281 | Chen, Liu, Xu |
| Illuminating the genome-wide activity of genome editors for safe and effective therapeutics | Genome Biology | Cheng et al. | 12/22/2018 | 30577870 | Tsai |
| Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer | Molecular Therapy: Methods & Clinical Development | Wang et al. | 12/14/2018 | 30397628 | Gao, Anderson, Xue |
| MicroRNA-96 Promotes Schistosomiasis Hepatic Fibrosis in Mice by Suppressing Smad7 | Molecular Therapy: Methods & Clinical Development | Luo et al. | 12/14/2018 | 30406154 | Gao, Anderson, Xue |
| Intracellular Delivery of His-Tagged Genome-Editing Proteins Enabled by Nitrilotriacetic Acid-Containing Lipidoid Nanoparticles | Advanced Healthcare Materials | Yamin et al. | 11/22/2018 | 30565897 | Chen, Liu, Xu |
| Targeting fidelity of adenine and cytosine base editors in mouse embryos | Nature Communications | Lee et al. | 11/15/2018 | 30442934 | Liu |
| Clinical Evaluations of Toxicity and Efficacy of Nanoparticle-Mediated Gene Therapy | Human Gene Therapy | Liang et al. | 11/13/2018 | 29893153 | Gao, Anderson, Xue |
| Understanding and repurposing CRISPR-mediated alternative splicing | Genome Biology | Smith et al. | 11/6/2018 | 30400804 | Gao, Anderson, Xue |
| Defining CRISPR–Cas9 genome-wide nuclease activities with CIRCLE-seq. | Nature Protocols | Lazzarotto et al. | 10/19/2018 | 30341435 | Tsai |
| Cas9-mediated Allelic Exchange Repairs Compound Heterozygous Recessive Mutations in Mice | Nature Biotechnology | Wang et al. | 10/1/2018 | 30102296 | Gao, Anderson, Xue |
| The temporal landscape of recursive splicing during Pol II transcription elongation in human cells | PLOS Genetics | Zhang et al. | 8/27/2018 | 30148885 | Gao, Anderson, Xue |
Phase 1 PublicationsAnne Temple2024-08-19T10:37:45-05:00