Generation of clinical grade human iPS cells
The therapeutic use of stem cells depends on the availability of pluripotent cells that are not limited by technical, ethical or immunological considerations. The goal of this proposal is to…
Derivation and characterization of human ES cells from FSHD embryos
Facioscapulohumeral muscular dystrophy (FSHD) is the third most common hereditary muscular dystrophy. It is autosomal dominant, meaning that if one of the parents has the disease, their children have a…
Using human embryonic stem cells to treat radiation-induced stem cell loss: Benefits vs cancer risk
A variety of stem cells exist in humans throughout life and maintain their ability to divide and change into multiple cell types. Different types of adult derived stem cells occur…
A modular automation approach to stem cell modeling to increase throughput, reproducibility and access
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.…
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…
Harnessing the rejuvenating capacity of pregnancy-associated factors to restore aged stem cell function
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…
Engineering AAV capsids for transduction of neural and muscle stem cells
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…
Drug Discovery for Duchenne Muscular Dystrophy Using Patient-Derived Human iPSCs
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…
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