Therapeutic/Technology: Exploring therapeutic Mechanism

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Interrogating Satellite Cell and Myofiber Defects and Repair in Human DMD using Single Nuclei/Single Cell RNA Sequencing of Muscle Resident Cells

Research Objective We will describe, for the first time, human muscle satellite cell, myofiber and immune cell dynamics due to dystrophin deficiency and AAV gene therapy in human muscle at single nuclei resolution. Impact These studies will elucidate satellite stem cell and myofiber defects in Duchenne and Becker Muscular Dystrophy and determine efficacy, mechanism and […]