Publications
Found 154 results
Type [ Year] Filters: %C2%A0vivo-gene-editing-muscle-stem-cells-adeno-associated-viral-vectors-mouse-model is [Clear All Filters]
AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities." Current Opinion in Biomedical Engineering 29 (2024).
"A humanized mouse model for adeno-associated viral gene therapy." Nat Commun 15, no. 1 (2024).
"Mechanosensitive genomic enhancers potentiate the cellular response to matrix stiffness." bioRxiv (2024).
"Multicenter integrated analysis of noncoding CRISPRi screens." Nat Methods (2024).
"An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair." Cell stem cell 30, no. 1 (2023): 96-111.e6.
"Nonviral In Vivo Delivery of CRISPR-Cas9 Using Protein-Agnostic, High-Loading Porous Silicon and Polymer Nanoparticles." ACS nano 17, no. 17 (2023): 16412-16431.
"Orthogonal CRISPR screens to identify transcriptional and epigenetic regulators of human CD8 T cell function." bioRxiv (2023).
"PCSK9 activation promotes early atherosclerosis in a vascular microphysiological system." APL bioengineering 7, no. 4 (2023).
"Transcriptional and epigenetic regulators of human CD8+ T cell function identified through orthogonal CRISPR screens." Nat Genet 55, no. 12 (2023): 2211-2223.
"Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing." Nat Commun 13, no. 1 (2022).
"Robust, Durable Gene Activation In Vivo via mRNA-Encoded Activators." ACS nano 16, no. 4 (2022): 5660-5671.
"AP-1 subunits converge promiscuously at enhancers to potentiate transcription." Genome Res 31, no. 4 (2021): 538-550.
"Branched-chain α-ketoacids are preferentially reaminated and activate protein synthesis in the heart." Nat Commun 12, no. 1 (2021).
"Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models." Nature communications 12, no. 1 (2021).
"Chromatin Remodeling of Colorectal Cancer Liver Metastasis is Mediated by an HGF-PU.1-DPP4 Axis." Adv Sci (Weinh) 8, no. 19 (2021).
"CRISPR Clocks: The Times They Are a-Changin'." The CRISPR journal 4, no. 2 (2021): 160-163.
"Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy." Mol Ther 29, no. 11 (2021): 3243-3257.
"Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science.. Vol. 23., 2021.
The NIH Somatic Cell Genome Editing program." Nature 592, no. 7853 (2021): 195-204.
"Transgenic mice for in vivo epigenome editing with CRISPR-based systems." Nat Methods 18, no. 8 (2021): 965-974.
"Enhancer RNAs predict enhancer-gene regulatory links and are critical for enhancer function in neuronal systems." Nucleic acids research 48, no. 17 (2020): 9550-9570.
"Immunity to Cas9 as an Obstacle to Persistent Genome Editing." Molecular therapy : the journal of the American Society of Gene Therapy 28, no. 6 (2020): 1389-1391.
"In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy." Mol Ther Methods Clin Dev 19 (2020): 320-329.
"Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation Screens." Cell Rep 33, no. 9 (2020).
"Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators." Stem cell reports 14, no. 5 (2020): 755-769.
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