| Publication Title | Authors | Journal | Pub Date | PubMed | Project PIs |
|---|---|---|---|---|---|
| Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens? | Wei Zhan, et al. | Frontiers in Immunology | 5/11/21 | 34046041 | Gao, Anderson, Xue |
| Sarcomere function activates a p53-dependent DNA damage response that promotes polyploidization and limits in vivo cell engraftment | Anthony M Pettinato, et al. | Cell Reports | 5/4/21 | 33951429 | Hinson |
| Targeting the Apoa1 locus for liver-directed gene therapy | De Giorgi, et al. | Molecular Therapy Methods & Clinical Development | 4/24/21 | TBD | Bao, Lagor |
| Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice | Liu P, et al. | Nature Communications | 4/9/21 | 33837189 | Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue |
| The NIH Somatic Cell Genome Editing program | Saha, K., Sontheimer, E.J., Brooks, P.J. et al. | Nature | 4/7/21 | 33828315 | SCGE Consortium |
| A CRISPR-Cas9-integrase complex generates precise DNA fragments for genome integration | Jakhanwal S, et al. | Nucleic Acids Research | 4/6/21 | 33693715 | Doudna, Banfield |
| Large-scale molecular epidemiological analysis of AAV in a cancer patient population | Qin W, et al. | Oncogene | 3/29/21 | 33782545 | Gao, Anderson, Xue |
| Nanoparticles for delivery of agents to fetal lungs | Ullrich SJ, et al. | Acta Biomaterialia | 3/15/21 | 33484911 | Saltzman, Glazer |
| Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3 | Qiu, Min, et al. | PNAS | 3/9/21 | 33649229 | Chen, Liu, Xu |
| Therapeutic RNA Delivery for COVID and Other Diseases | Dobrowolski C, et al. | Advanced Healthcare Materials | 3/4/21 | 33661555 | Dahlman, Santangelo |
| Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy | Marquez Loza LI, et al. | Molecular Therapy Methods & Clinical Development | 2/27/21 | 33768133 | McCray |
| Tissue-Engineered Skeletal Muscle Models to Study Muscle Function, Plasticity, and Disease. | Khodabukus A. | Frontiers in Physiology | 2/26/21 | 33716768 | Gersbach, Bursac, Truskey |
| COVID-19 CG enables SARS-CoV-2 mutation and lineage tracking by locations and dates of interest | Chen AT, et al. | Elife | 2/23/21 | 33620031 | Deverman |
| Precision genome editing using cytosine and adenine base editors in mammalian cells | Huang, TP, et al. | Nature Protocols | 2/16/21 | 33462442 | Liu |
| PrimeDesign software for rapid and simplified design of prime editing guide RNAs | Hsu, Jonathan Y, et al. | Nature Communications | 2/15/21 | 33589617 | Liu |
| Soluble CD40L activates soluble and cell-surface integrin αvβ3, α5β1, and α4β1 by binding to the allosteric ligand-binding site (site 2) | Takada YK, et al. | Journal of Biological Chemistry | 2/9/21 | 33571526 | Lam, Cheng |
| Viral vector platforms within the gene therapy landscape. | Bulcha, Jote T, et al. | Signal Transduction and Targeted Therapy | 2/8/21 | 33558455 | Gao, Anderson, Xue |
| Gene-based therapies for neurodegenerative diseases | Sun, Jichao, et al | Nature Neuroscience | 2/1/21 | 33526943 | Gong, Emborg, Levine, Roy, Saha |
| Protein-based nanoplatform for detection of tumorigenic polyps in the colon via noninvasive mucosal routes | Chen, CC, et al. | Pharmaceutical Patent Analysis | 1/20/21 | 33467938 | Lam, Cheng |
| Engineering highly efficient backsplicing and translation of synthetic circRNAs | Meganck, RM, et al. | Molecular Therapy - Nucleic Acids Journal | 1/16/21 | 336142328 | Asokan |
| Tools for experimental and computational analyses of off-target editing by programmable nucleases | Bao, X Robert, et al. | Nature Protocols | 1/16/21 | 33288953 | Bao |
| In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice. | Kloban, AL, et al. | Nature | 1/6/21 | 33408413 | Liu |
| Overcoming innate immune barriers that impede AAV gene therapy vectors | Muhuri, M, et al. | The Journal of Clinical Investigation | 1/4/21 | 33393506 | Gao, Anderson, Xue |
| Frame-Hydrogel Methodology for Engineering Highly Functional Cardiac Tissue Constructs | Heifer, Abbigail, et al. | Methods in Molecular Biology | 1/1/21 | 32857373 | Gersbach, Bursac, Truskey |
| Targeting de novo purine synthesis pathway via ADSL depletion impairs liver cancer growth by perturbing mitochondrial function | Jiang, Tingting, et al. | Hepatology | 12/17/20 | 33336367 | Gao, Anderson, Xue |
| Multicellular Systems to Translate Somatic Cell Genome Editors to Humans | Hernandez-Gordillo V, et al. | Current Opinion in Biotechnology | 12/16/20 | 33718690 | Kiana |
| The dawn of non-human primate models for neurodevelopmental disorders. | Aida, Tomoni, et al.. | Current Opinion in Genetics and Development | 12/1/20 | 33718690 | Feng |
| Advances in microphysiological blood-brain barrier (BBB) models towards drug delivery | Lee, Caleb S, et al. | Current Opinion in Biotechnology | 12/1/20 | 32711361 | Leong |
| Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation Screens | Black, Joshua B, et al. | Cell Reports | 12/1/20 | 33264623 | Gersbach |
| Poison Exon Splicing Regulates a Coordinated Network of SR Protein Expression during Differentiation and Tumorigenesis. | Leclair, Nathan K, et al. | Molecular Cell | 11/19/20 | 33176162 | Hinson |
| Phage-assisted continuous and non-continuous evolution | Miller, Shannon M, et al. | Nature Protocols | 11/16/20 | 33199872 | Liu |
| The once and future gene therapy | Bulaklak, Karen, et al. | Nature Communications | 11/16/20 | 33199717 | Gersbach, Bursac, Truskey | Gersbach |
| Delayed urea differential enhancement CEST (dudeCEST)-MRI with T 1 correction for monitoring renal urea handling | Shin, Soo Hyun, et al. | Magnetic Resonance in Medicine | 11/12/20 | 33180343 | Vandsberger |
| Advances in Alpha-1 Antitrypsin Gene Therapy | Lorincz, Reka, et al. | America Journal of Respiratory Cell and Nolecular Biology | 11/1/20 | 32668173 | Curiel |
| The Golgi Calcium ATPase Pump Plays an Essential Role in Adeno-associated Virus Trafficking and Transduction. | Madigan, Victoria J, et al. | Journal of Virology | 10/14/20 | 32817219 | Asokan |
| A Versatile Nonviral Delivery System for Multiplex Gene-Editing in the Liver | Gong, Jing, et al. | Advanced Materials | 10/14/20 | 33053221 | Leong |
| 3D kidney organoids for bench-to-bedside translation | Gupta, Navin, et al. | Journal of Molecular Medicine | 10/9/20 | 33034708 | Morizane, Lewis, Sabbisetti |
| Precision Cas9 Genome Editing in vivo with All-in-one, Self-targeting AAV Vectors | Ibraheim, Raed, et al. | bioRxiv | 10/9/20 | Preprint | Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue |
| Development of a Cardiac Sarcomere Functional Genomics Platform to Enable Scalable Interrogation of Human TNNT2 Variants | Pettinato, Anthony M, et al. | Circulation | 10/7/20 | 33025817 | Hinson |
| Opportunities and limitations of genetically modified nonhuman primate models for neuroscience research | Feng, Guoping, et al. | Proceeding of the National Academy of Sciences in the United States of America | 9/29/20 | 32817435 | Feng |
| In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy | Kwon, Jennifer B, et al. | Molecular Therapy Methods & Clinical Development | 9/28/20 | 33145368 | Asokan | Gersbach, Bursac, Truskey |
| COVID-19 CG:Tracking SARS-CoV-2 mutations by locations and dates of interest | Chen, Albert Tian, et al. | bioRxiv | 9/28/20 | 32995794 | Deverman |
| Synthetic immunomodulation with a CRISPR super-repressor in vivo | Moghadam, Farzaneh, et al. | Nature Cell Biology | 9/22/20 | 32884147 | Kiani |
| Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach | Havalik, Patrick L, et al. | Journal of Virology | 9/15/20 | 32669336 | Asokan |
| SARS-CoV-2 infection of human iPSC-derived cardiac cells predicts novel cytopathic features in hearts of COVID-19 patients | Pérez-Bermejo, Juan A, et al. | bioRxiv | 9/12/20 | 32884147 | McDevitt |
| Direct Functional Protein Delivery with a Peptide into Neonatal and Adult Mammalian Inner Ear In Vivo | Zhang, Kun, et al. | Molecular Therapy Methods & Clinical Development | 9/11/20 | 32953927 | Chen, Liu, Xu |
| Rapid, precise quantification of large DNA excisions and inversions by ddPCR. | Watry, Hannah L, et al. | Scientific Reports | 9/10/20 | 32913194 | McDevitt |
| A pH-responsive silica-metal-organic framework hybrid nanoparticle for the delivery of hydrophilic drugs, nucleic acids, and CRISPR-Cas9 genome-editing machineries. | Wang, Yuyuan, et al. | Journal of Controlled Release | 8/10/20 | 32380204 | Gong, Emborg, Levine, Roy, Saha |
| A scoutRNA Is Required for Some Type V CRISPR-Cas Systems | Harrington, Lucas B, et al. | Molecular Cell | 8/6/20 | 32645367 | Doudna, Banfield |
| NDAT Targets PI3K-Mediated PD-L1 Upregulation to Reduce Proliferation in Gefitinib-Resistant Colorectal Cancer. | Huang, Tung-Yung, et al. | Cells | 8/3/20 | 32756527 | Lam, Cheng |
| Finding order in slow axonal transport | Wang, Yuyuan, et al. | Current Opinion in Neurobiology | 8/1/20 | 32361600 | Gong, Emborg, Levine, Roy, Saha |
| DNA capture by a CRISPR-Cas9-guided adenine base editor. | Al-Shayeb, Basem, et al. | Science | 7/31/20 | 32732424 | Doudna, Banfield | Liu |
| Epigenetic transcriptional reprogramming by WT1 mediates a repair response during podocyte injury. | Ettou, Sandrine, et al. | Science Advances | 7/24/20 | 32754639 | Morizane, Lewis, Sabbisetti |
| Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning. | Arbab, Mandana, et al. | Cell | 7/23/20 | 32533916 | Liu |
| CRISPR-CasΦ from huge phages is a hypercompact genome editor. | Pausch, Patrick, et al. | Science | 7/17/20 | 32675376 | Doudna, Banfield |
| Combinatorial Library of Cyclic Benzylidene Acetal-Containing pH-Responsive Lipidoid Nanoparticles for Intracellular mRNA Delivery. | Li, Yamin, et al. | Bioconjugate Chemistry | 7/15/20 | 32520527 | Chen, Liu, Xu |
| Tissue-Engineered Human Myobundle System as a Platform for Evaluation of Skeletal Muscle Injury Biomarkers. | Khodabukus, Alastair, et al. | Toxicological Sciences | 7/1/20 | 32294208 | Gersbach, Bursac, Truskey |
| The delivery challenge: fulfilling the promise of therapeutic genome editing | van Haasteren, Joost, et al. | Nature Biotechnology | 6/29/20 | 32601435 | Bankiewicz, Murthy |
| Programmable m6A modification of cellular RNAs with a Cas13-directed methyltransferase. | Wilson, Christopher, et al. | Nature Biotechnology | 6/29/20 | 32601430 | Liu |
| Polymeric vehicles for nucleic acid delivery. | Piotrowski-Daspit, Alexandra S, et al. | Advance Drug Delivery Reviews | 6/23/20 | 32585159 | Saltzman, Glazer |
| Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors. | Anzalone, Andrew V, et al. | Nature Biotechnology | 6/22/20 | 32572269 | Liu |
| A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing. | Mok, Beverly Y, et al. | Nature | 6/22/20 | 32641830 | Liu |
| CHANGE-seq reveals genetic and epigenetic effects on CRISPR-Cas9 genome-wide activity. | Lazzarotto, Cicera R, et al. | Nature Biotechnology | 6/15/20 | 32541958 | Tsai |
| Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells. | Nagpal, Neha, et al. | Cell Stem Cell | 6/4/20 | 32320679 | Sontheimer, Khvorova, Watts, Wolfe |
| AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9. | Li, Ang, et al. | Molecular Therapy | 6/3/20 | 32348718 | Bao, Lagor |
| Can we mimic skeletal muscles for novel drug discovery? | Broer, Torie, et al. | Expert Opinion on Drug Delivery | 6/1/20 | 32122176 | Gersbach, Bursac, Truskey |
| Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligands | Gan Z, et al. | Bioengineering & Translational Medicine | 5/27/20 | 33758781 | Dahlman, Santangelo |
| YAP1 withdrawal in hepatoblastoma drives therapeutic differentiation of tumor cells to functional hepatocyte-like cells. | Smith, Jordan L, et al. | Hepatology | 5/26/20 | 32452550 | Gao, Anderson, Xue | Liu |
| The developing toolkit of continuous directed evolution | Morrison, Mary S, et al. | Nature Chemical Biology | 5/22/20 | 32444838 | Liu |
| Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors | Rumachik, Neil G, et al. | Molecular Therapy, Methods & Clinical Development | 5/22/20 | 32995354 | McDevitt |
| Protein and mRNA Delivery Enabled by Cholesteryl-Based Biodegradable Lipidoid Nanoparticles. | Li, Yamin, et al. | Angewandte Chemie International Edition in English | 5/21/20 | 32438474 | Chen, Liu, Xu |
| Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators | Kwon, Jennifer B, et al. | Stem Cell Reports | 5/12/20 | 32330446 | Gersbach, Bursac, Truskey | Asokan | Gersbach |
| Advances in genome editing for genetic hearing loss. | Ding, Ning, et al. | Advance Drug Delivery Reviews | 5/7/20 | 32387678 | Bao, Lagor |
| Once upon a dish: engineering multicellular systems | Haase, Kristina, et al. | Development | 5/4/20 | 32366376 | Freedman |
| Positron emission tomography imaging of novel AAV capsids maps rapid brain accumulation | Seo, Jai Woong, et al. | Nature Communications | 4/30/20 | 32355221 | Lam, Cheng |
| Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope. | Jiang, Tingting, et al. | Nature Communications | 4/24/20 | 32332735 | Gao, Anderson, Xue | Liu |
| Prime genome editing in rice and wheat | Lin, Qiupeng, et al. | Nature Biotechnology | 3/16/20 | 32393904 | Liu |
| Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity. | Richter, Michelle F, et al. | Nature Biotechnology | 3/16/20 | 32433547 | Liu |
| Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases | Wu, Shao-Shuai, et al. | Theranostics | 3/15/20 | 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 | Zhou, Yu, et al. | Advanced Science | 3/7/20 | 32154067 | Zhou |
| Profiling APOL1 Nephropathy Risk Variants in Genome-Edited Kidney Organoids with Single-Cell Transcriptomics. | Liu, Esther, et al. | Kidney360 | 3/1/20 | 32656538 | Freedman |
| Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications | Li, Yamin, et al. | Biomaterials | 3/1/20 | 31945616 | Chen, Liu, Xu |
| Adenine base editing in an adult mouse model of tyrosinaemia. | Song, Chun-Qing, et al. | Nature Biomedical Engineering | 2/25/20 | 31740768 | Liu | Gao, Anderson, Xue |
| Poly(Lactic-co-Glycolic Acid) Nanoparticle Delivery of Peptide Nucleic Acids In Vivo. | Oyaghire, Stanley N, et al. | Methods in Molecular Biology | 2/23/20 | 32088877 | Saltzman, Glazer |
| High-throughput quantitative microscopy-based half-life measurements of intravenously injected agents | Bracaglia, Laura G, et al. | Proceeding of the National Academy of Sciences in the United States of America | 2/18/20 | 32015123 | Saltzman, Glazer |
| Site-Specific Post-translational Surface Modification of Adeno-Associated Virus Vectors Using Leucine Zippers | Thadani, Nicole N, et al. | ACS Synthetic Biology | 2/18/20 | 32068391 | Bao, Lagor |
| Clades of huge phages from across Earth's ecosystems | Al-Shayeb, Basem, et al. | Nature | 2/12/20 | 32051592 | Doudna, Banfield |
| Quantitating Endosomal Escape of a Library of Polymers for mRNA Delivery | Jiang, Yuhang, Lu, et al. | Nano Letters | 2/12/20 | 32003222 | Saltzman, Glazer |
| Continuous evolution of SpCas9 variants compatible with non-G PAMs. | Miller, Shannon M, et al. | Nature Biotechnology | 2/10/20 | 32042170 | Liu |
| Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors. | Doman, Jordan L, et al. | Nature Biotechnology | 2/10/20 | 32042165 | Liu |
| Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair. | Economos, Nicholas G, et al. | Molecules | 2/8/20 | 32046275 | Saltzman, Glazer | Glazer, Ly, Saltzman |
| Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs. | Osborn, Mark J, et al. | The Journal of Investigative Dermatology | 2/1/20 | 31437443 | Liu |
| From GWAS Association to Function: Candidate Gene Screening Within Insulin Resistance-Associated Genomic Loci Using a Preadipocyte Differentiation Model | Ladha, Feria A, et al. | Circulation Research | 1/31/20 | 31999535 | Hinson |
| Detection of Extracellular Vesicle RNA Using Molecular Beacons. | Oliveira Jr, Getulio Pereira de, et al. | iScience | 1/24/20 | 31958756 | Ghiran |
| Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses. | Levy, JM, et al. | Nature Biomedical Engineering | 1/4/20 | 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. | Maynard, Lucy H, et al. | Human Gene Therapy Methods | 12/19/19 | 31855083 | McDevitt |
| Structures of Neisseria meningitidis Cas9 Complexes in Catalytically Poised and Anti-CRISPR-Inhibited States. | Sun, W, et al. | Molecular Cell | 12/19/19 | 31668930 | Sontheimer, Khvorova, Watts, Wolfe |
| Optimizing biodegradable nanoparticle size for tissue-specific delivery. | Mandl, HK, et al. | Journal of Controlled Release | 11/28/19 | 31654688 | Saltzman, Glazer |
| Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids | Huang, Qin, et al. | PLOS ONE | 11/14/19 | 31725765 | Deverman |
| Genome editing of HBG1 and HBG2 to induce fetal hemoglobin | Métais, Jean-Yves, et al. | Blood Advances | 11/12/19 | 31698466 | Tsai |
| Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia | Sateesh Krishnamurthy, et al. | Nature Communications | 10/28/19 | 31659165 | McCray |
| Search-and-replace genome editing without double-strand breaks or donor DNA | Anzalone AV, et al. | Nature | 10/21/19 | 31634902 | Liu |
| All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo. | Ibraheim, Raed, et al. | Genome Biology | 9/19/19 | 30231914 | Gao, Anderson, Xue |
| A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing | Chen G, et al. | Nature Nanotechnology | 9/9/19 | 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? | Qiu, Min, et al. | Biomacromolecules | 9/9/19 | 31342740 | Chen, Liu, Xu |
| High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs. | Xu, Meiyu, et al. | Human Gene Therapy | 8/30/19 | 31072208 | Gao, Anderson, Xue |
| Mapping cis-regulatory chromatin contacts in neural cells links neuropsychiatric disorder risk variants to target genes. | Song, Michael, et al. | Nature Genetics | 7/31/19 | 31367015 | McDevitt |
| Continuous evolution of base editors with expanded target compatibility and improved activity | Thuronyi BW, et al. | Nature Biotechnology | 7/22/19 | 31332326 | Liu |
| Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis. | Yang, Yeon-Suk, et al. | Nature Communications | 7/4/19 | 31273195 | Gao, Anderson, Xue |
| An anionic human protein mediates cationic liposome delivery of genome editing proteins into mammalian cells | Kim, Y Bill, et al. | Nature Communications | 7/2/19 | 31266953 | Liu |
| Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles. | Liu, J, et al. | Advanced Materials | 6/19/19 | 31215123 | Chen, Liu, Xu |
| Depletion of TRRAP Induces p53-Independent Senescence in Liver Cancer by Down-Regulating Mitotic Genes. | Kwan, Suet-Yan, et al. | Hepatology | 6/12/19 | 31188495 | Gao, Anderson, Xue |
| Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors. | Huang, Tony P, et al. | Nature Biotechnology | 6/10/19 | 31110355 | Liu |
| Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity | Xiao, Yuanyuan, et al. | JCI Insight | 5/21/19 | 31112525 | Gao, Anderson, Xue |
| Adeno-associated virus vector as a platform for gene therapy delivery. | Wang D, et al. | Nature Reviews Drug Discovery | 5/18/19 | 30710128 | Gao, Anderson, Xue |
| Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks. | Rees, Holly A, et al. | Nature Communications | 5/17/19 | 31101808 | Liu |
| Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy | Gong, Yi, et al. | Human Gene Therapy | 5/15/19 | 30358470 | Gao, Anderson, Xue |
| Analysis and minimization of cellular RNA editing by DNA adenine base editors | Rees HA, et al. | Science Advances | 5/8/19 | 31086823 | Liu |
| Nucleic Acid Therapeutics for Neurological Diseases | Watts, Jonathan K, et al. | Neurotherapeutics | 4/29/19 | 31037650 | Sontheimer, Khvorova, Watts, Wolfe |
| Unbiased detection of CRISPR off-targets in vivo using DISCOVER-Seq | Wienert, Beeke, et al. | Science | 4/19/19 | 31000663 | McDevitt |
| CRISPR-SONIC: targeted somatic oncogene knock-in enables rapid in vivo cancer modeling. | Mou, Haiwei, et al. | Genome Medicine | 4/16/19 | 30987660 | Gao, Anderson, Xue |
| An orange calcium-modulated bioluminescent indicator for non-invasive activity imaging. | Oh, Younghee, et al. | Nature Chemical Biology | 4/1/19 | 30936501 | McDevitt |
| Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression. | Martinez-Navio, et al. | Immunity | 3/19/19 | 30850342 | Gao, Anderson, Xue |
| Anti-drug Antibody Responses Impair Prophylaxis Mediated by AAV-Delivered HIV-1 Broadly Neutralizing Antibodies | Gardner, Matthew R, et al. | Molecular Therapy | 3/6/19 | 30704961 | Gao, Anderson, Xue |
| A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing | Edraki, Alireza, et al. | Molecular Cell | 2/21/19 | 30581144 | Gao, Anderson, Xue |
| Enhancing the In Vitro and In Vivo Stabilities of Polymeric Nucleic Acid Delivery Nanosystems. | Wang, Yuyuan, et al. | Bioconjugate Chemistry | 2/20/19 | 30592619 | Gong, Emborg, Levine, Roy, Saha |
| Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System | Gessler, Dominic J, et al. | Methods in Molecular Biology | 2/20/19 | 30783972 | Gao, Anderson, Xue |
| Quantitative and Digital Droplet-Based AAV Genome Titration | Sanmiguel, Julio, et al. | Methods in Molecular Biology | 2/20/19 | 30783968 | Gao, Anderson, Xue |
| Simultaneous targeting of linked loci in mouse embryos using base editing | Lee, Hye Kyung, et al. | Scientific Reports | 2/7/19 | 30733567 | Liu |
| Integrating Combinatorial Lipid Nanoparticle and Chemically Modified Protein for Intracellular Delivery and Genome Editing | Chang, Jin, et al. | Accounts of Chemical Research | 12/26/18 | 30586281 | Chen, Liu, Xu |
| Illuminating the genome-wide activity of genome editors for safe and effective therapeutics | Cheng, Yong, et al. | Genome Biology | 12/22/18 | 30577870 | Tsai |
| Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer | Wang, Dan, et al. | Molecular Therapy: Methods & Clinical Development | 12/14/18 | 30397628 | Gao, Anderson, Xue |
| MicroRNA-96 Promotes Schistosomiasis Hepatic Fibrosis in Mice by Suppressing Smad7 | Luo, Xufeng, et al. | Molecular Therapy: Methods & Clinical Development | 12/14/18 | 30406154 | Gao, Anderson, Xue |
| Intracellular Delivery of His-Tagged Genome-Editing Proteins Enabled by Nitrilotriacetic Acid-Containing Lipidoid Nanoparticles | Yamin Li, et al. | Advanced Healthcare Materials | 11/22/18 | 30565897 | Chen, Liu, Xu |
| Targeting fidelity of adenine and cytosine base editors in mouse embryos | Lee, Hye Kyung, et al. | Nature Communications | 11/15/18 | 30442934 | Liu |
| Clinical Evaluations of Toxicity and Efficacy of Nanoparticle-Mediated Gene Therapy | Liang, Xiao, et al. | Human Gene Therapy | 11/13/18 | 29893153 | Gao, Anderson, Xue |
| Understanding and repurposing CRISPR-mediated alternative splicing | Smith, Jordan L, et al. | Genome Biology | 11/6/18 | 30400804 | Gao, Anderson, Xue |
| Defining CRISPR–Cas9 genome-wide nuclease activities with CIRCLE-seq. | Lazzarotto, Cicera R, et al. | Nature Protocols | 10/19/18 | 30341435 | Tsai |
| Cas9-mediated Allelic Exchange Repairs Compound Heterozygous Recessive Mutations in Mice | Wang, Dan, et al. | Nature Biotechnology | 10/1/18 | 30102296 | Gao, Anderson, Xue |
| The temporal landscape of recursive splicing during Pol II transcription elongation in human cells | Zhang, Xiao-Ou, et al. | PLOS Genetics | 8/27/18 | 30148885 | Gao, Anderson, Xue |
PUBLICATIONSAnne Temple2021-03-17T15:04:58-05:00