This project enhances stem cell access, scalability, and collaboration. It offers characterized hPSC lines, CRISPR editing, and differentiation on automated platforms accelerating progress in biology, disease research, and regenerative medicine.…
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…
Research Objective Elucidation of pregnancy-related factors that mitigate cellular senescence and enhance regeneration has far-reaching implications for understanding the mechanisms of aging and rejuvenation. Impact The study will address the…
Research Objective The studies will identify and characterize new gene therapy vectors able to deliver gene editing components to stem cells. to enable treatment of diseases involving both muscle and…
Research Objective We will utilize human induced pluripotent stem cells derived from Duchenne muscular dystrophy (DMD) patients for drug testing and drug discovery for this rare genetic disease. Impact Diverse…
Research Objective To develop a lipid nanoparticle/mRNA complex that can safely and efficiently edit muscle stem cells in utero, correct the dystrophin mutation, and develop a treatment for Duchenne muscular…
Research Objective Conditioned media from human iPSC-derived smooth muscle cell progenitors. This media exerts paracrine effect to restore damaged vaginal wall in patients with pelvic organ prolapse. Impact Pelvic organ…
Research Objective We seek to develop a cell based-hydrogel therapy to improve outcomes in patients with muscle degeneration. The technology will improve muscle through sustained release of cell-based cytokines. Impact…
Research Objective We will 1) identify non-invasive bladder cancer patients with (pre)cancerous urothelium by single-cell RNA-seq and 2) replace this dangerous lesion with normal hESC-derived bladder progenitors. Impact Replacement of…
Research Objective We will molecularly and functionally define muscle stem cells in human muscle in development, juvenile and adult and develop strategies to generate the most regenerative muscle stem cells…
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