Publication Title | Journal | Authors | Pub Date | PubMed | Project PIs |
---|---|---|---|---|---|
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 |
CRISPR-Cas9 bends and twists DNA to read its sequence | Nature Structural & Molecular Biology | Cofsky et al | 4/29/2022 | 35422516 | Doudna |
Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome Editing | ACS Publications | Chen et al | 4/21/2022 | 35446558 | Sontheimer, Khvorova, Watts, Wolfe |
Crystal structure of an RNA/DNA strand exchange junction | PLOS One | Cofsky et al | 4/18/2022 | 35436289 | Doudna |
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 |
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 | Xue, Sontheimer |
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 |
A self-immolative linker that releases thiols detects penicillin amidase and nitroreductase with high sensitivity via absorption spectroscopy | ChemComm | Espinoza et al | 3/3/2022 | 35170593 | 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 | 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 Review | 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 |
AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies | Trends in Molecular Medicine | Yang et al | 2/12/2022 | 33714697 | Gao |
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 |
Gene editing in a Myo6 semi-dominant mouse model rescues auditory function | Molecular Therapy | Xue et al | 1/5/2022 | 34174443 | Chen |
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 | Wolfe, Xue |
pH-Responsive Polymer Nanoparticles for Efficient Delivery of Cas9 Ribonucleoprotein With or Without Donor DNA | Advanced Materials | Xie et al | 2/4/2022 | 35119139 | Gong |
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 |
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins | Cell | Banskota et al | 1/20/2022 | 35021064 | Liu | Chaikof |
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 |
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 |
Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical Applications | Materials Today (Kidlington England) | Hu H et al | 1/1/2022 | 34421329 | Leong |
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 |
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 Y et al | 12/1/2021 | 34483808 | Leong |
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 |
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 M 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 |
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 T, 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 S, 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 |
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 P, et al. | 8/28/2021 | 34381246 | Doudna, Banfield |
Easy-Prime: a machine learning-based prime editor design tool | Genome Biology | Li et al. | 8/19/2022 | 34412673 | Tsai |
Transgenic mice for in vivo epigenome editing with CRISPR-based systems | Nature Methods | Gemberling et al. | 8/18/2021 | 34341582 | Gersbach | Asokan |
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. | 8/10/2021 | 32380204 | Gong, Emborg, Levine, Roy, Saha |
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 |
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 GA, et al. | 7/1/2021 | 34079130 | Liu | Tsai |
Mucopolysaccharidoses type I gene therapy | Journal of Inherited Metabolic Disease | Hurt, SC | 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 LW, et al. | 6/28/2021 | 34183861 | Liu | Chaikof |
Induced pluripotent stem cells provide mega insights into kidney disease | Kidney International | Luciani A, 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 LI, et al. | 6/11/2021 | 33768133 | McCray |
Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repair | Cell | Wilde JJ, 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 J, et al. | 6/1/2021 | 34075401 | Yi |
Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering | Cell Reports | Hamilton JR, 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 X, et al | 5/25/2021 | 34070435 | Chen, Liu, Xu |
Quantification of Cellular Densities and Antigenic Properties using Magnetic Levitation | Journal of Visualized Experiements | Thompson L, 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 | Wei Zhan, et al. | 5/11/2021 | 34046041 | Gao, Anderson, Xue |
Sarcomere function activates a p53-dependent DNA damage response that promotes polyploidization and limits in vivo cell engraftment | Cell Reports | Anthony M 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 JJ, 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 H, 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 |
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 P, 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 S, 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, Neil G, et al. | 4/1/2021 | 33868302 | McDevitt |
Large-scale molecular epidemiological analysis of AAV in a cancer patient population | Oncogene | Qin W, 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 |
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 JA, et al. | 3/15/2021 | 33723017 | McDevitt |
Nanoparticles for delivery of agents to fetal lungs | Acta Biomaterialia | Ullrich SJ, et al. | 3/15/2021 | 33484911 | Saltzman, Glazer |
Therapeutic RNA Delivery for COVID and Other Diseases | Advanced Healthcare Materials | Dobrowolski C, 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 |
Tissue-Engineered Skeletal Muscle Models to Study Muscle Function, Plasticity, and Disease. | Frontiers in Physiology | Khodabukus A. | 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 AT, 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, TP, et al. | 2/16/2021 | 33462442 | Liu |
PrimeDesign software for rapid and simplified design of prime editing guide RNAs | Nature Communications | Hsu, Jonathan Y, 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 YK, et al. | 2/9/2021 | 33571526 | Lam, Cheng |
Viral vector platforms within the gene therapy landscape. | Signal Transduction and Targeted Therapy | Bulcha, Jote T, et al. | 2/8/2021 | 33558455 | Gao, Anderson, Xue |
Gene-based therapies for neurodegenerative diseases | Nature Neuroscience | Sun, Jichao, 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 JJ, 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, CC, et al. | 1/20/2021 | 33467938 | Lam, Cheng |
Engineering highly efficient backsplicing and translation of synthetic circRNAs | Molecular Therapy - Nucleic Acids Journal | Meganck, RM, et al. | 1/16/2021 | 336142328 | Asokan |
Tools for experimental and computational analyses of off-target editing by programmable nucleases | Nature Protocols | Bao, X Robert, et al. | 1/16/2021 | 33288953 | Bao |
Exercise mimetics and JAK inhibition attenuate IFN-γ-induced wasting in engineered human skeletal muscle | Science Advances | Chen Z, et al. | 1/7/2021 | 33523949 | Gersbach, Bursac, Truskey |
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice. | Nature | Kloban, AL, et al. | 1/6/2021 | 33408413 | Liu |
Overcoming innate immune barriers that impede AAV gene therapy vectors | The Journal of Clinical Investigation | Muhuri, M, et al. | 1/4/2021 | 33393506 | Gao, Anderson, Xue |
Frame-Hydrogel Methodology for Engineering Highly Functional Cardiac Tissue Constructs | Methods in Molecular Biology | Heifer, Abbigail, et al. | 1/1/2021 | 32857373 | Gersbach, Bursac, Truskey |
Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical Applications | Mater Today (Kidlington) | Hu H, et al. | 1/1/2021 | 34421329 | Leong |
Targeting de novo purine synthesis pathway via ADSL depletion impairs liver cancer growth by perturbing mitochondrial function | Hepatology | Jiang, Tingting, 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 V, et al. | 12/16/2020 | 33718690 | Kiana |
The dawn of non-human primate models for neurodevelopmental disorders. | Current Opinion in Genetics and Development | Aida, Tomoni, et al.. | 12/1/2020 | 33718690 | Feng |
Advances in microphysiological blood-brain barrier (BBB) models towards drug delivery | Current Opinion in Biotechnology | Lee, Caleb S, 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, Joshua B, 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, Nathan K, et al. | 11/19/2020 | 33176162 | Hinson |
Phage-assisted continuous and non-continuous evolution | Nature Protocols | Miller, Shannon M, et al. | 11/16/2020 | 33199872 | Liu |
The once and future gene therapy | Nature Communications | Bulaklak, Karen, 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, Soo Hyun, et al. | 11/12/2020 | 33180343 | Vandsburger |
Advances in Alpha-1 Antitrypsin Gene Therapy | America Journal of Respiratory Cell and Nolecular Biology | Lorincz, Reka, 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, Victoria J, et al. | 10/14/2020 | 32817219 | Asokan |
A Versatile Nonviral Delivery System for Multiplex Gene-Editing in the Liver | Advanced Materials | Gong, Jing, et al. | 10/14/2020 | 33053221 | Leong |
3D kidney organoids for bench-to-bedside translation | Journal of Molecular Medicine | Gupta, Navin, et al. | 10/9/2020 | 33034708 | Morizane, Lewis, Sabbisetti |
Precision Cas9 Genome Editing in vivo with All-in-one, Self-targeting AAV Vectors | bioRxiv | Ibraheim, Raed, et al. | 10/9/2020 | Preprint | Sontheimer, Khvorova, Watts, Wolfe | Gao, Anderson, Xue |
Development of a Cardiac Sarcomere Functional Genomics Platform to Enable Scalable Interrogation of Human TNNT2 Variants | Circulation | Pettinato, Anthony M, 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, Guoping, et al. | 9/29/2020 | 32817435 | Feng |
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, Jennifer B, et al. | 9/28/2020 | 33145368 | Asokan | Gersbach, Bursac, Truskey |
COVID-19 CG:Tracking SARS-CoV-2 mutations by locations and dates of interest | bioRxiv | Chen, Albert Tian, et al. | 9/28/2020 | 32995794 | Deverman |
Synthetic immunomodulation with a CRISPR super-repressor in vivo | Nature Cell Biology | Moghadam, Farzaneh, et al. | 9/22/2020 | 32884147 | Kiani |
Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach | Journal of Virology | Havalik, Patrick L, 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, Juan A, 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, Kun, et al. | 9/11/2020 | 32953927 | Chen, Liu, Xu |
Rapid, precise quantification of large DNA excisions and inversions by ddPCR. | Scientific Reports | Watry, Hannah L, et al. | 9/10/2020 | 32913194 | McDevitt |
New Directions in Pulmonary Gene Therapy | Human Gene Therapy | Vu, A et al. | 9/3/2020 | 32814451 | McCray |
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, Yuyuan, 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, Lucas B, 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, Tung-Yung, et al. | 8/3/2020 | 32756527 | Lam, Cheng |
Finding order in slow axonal transport | Current Opinion in Neurobiology | Wang, Yuyuan, et al. | 8/1/2020 | 32361600 | Gong, Emborg, Levine, Roy, Saha |
DNA capture by a CRISPR-Cas9-guided adenine base editor. | Science | Al-Shayeb, Basem, et al. | 7/31/2020 | 32732424 | Doudna, Banfield | Liu |
Epigenetic transcriptional reprogramming by WT1 mediates a repair response during podocyte injury. | Science Advances | Ettou, Sandrine, et al. | 7/24/2020 | 32754639 | Morizane, Lewis, Sabbisetti |
Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning. | Cell | Arbab, Mandana, et al. | 7/23/2020 | 32533916 | Liu |
CRISPR-CasΦ from huge phages is a hypercompact genome editor. | Science | Pausch, Patrick, 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, Yamin, 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, Alastair, et al. | 7/1/2020 | 32294208 | Gersbach, Bursac, Truskey |
The delivery challenge: fulfilling the promise of therapeutic genome editing | Nature Biotechnology | van Haasteren, Joost, et al. | 6/29/2020 | 32601435 | Bankiewicz, Murthy |
Programmable m6A modification of cellular RNAs with a Cas13-directed methyltransferase. | Nature Biotechnology | Wilson, Christopher, et al. | 6/29/2020 | 32601430 | Liu |
Polymeric vehicles for nucleic acid delivery. | Advance Drug Delivery Reviews | Piotrowski-Daspit, Alexandra S, et al. | 6/23/2020 | 32585159 | Saltzman, Glazer |
Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors. | Nature Biotechnology | Anzalone, Andrew V, et al. | 6/22/2020 | 32572269 | Liu |
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing. | Nature | Mok, Beverly Y, et al. | 6/22/2020 | 32641830 | Liu |
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Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis. | Nature Communications | Yang, Yeon-Suk, et al. | 7/4/2019 | 31273195 | Gao, Anderson, Xue |
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CRISPR-SONIC: targeted somatic oncogene knock-in enables rapid in vivo cancer modeling. | Genome Medicine | Mou, Haiwei, et al. | 4/16/2019 | 30987660 | Gao, Anderson, Xue |
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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 |
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MicroRNA-96 Promotes Schistosomiasis Hepatic Fibrosis in Mice by Suppressing Smad7 | Molecular Therapy: Methods & Clinical Development | Luo, Xufeng, et al. | 12/14/2018 | 30406154 | Gao, Anderson, Xue |
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PUBLICATIONSAnne Temple2022-01-06T12:55:07-05:00