Research Objective The therapeutic candidate proposed here is hematopoietic stem/progenitor cells engineered to encode for HIV-specific T cell receptors. Impact The success of the proposed studies will test the efficacy…
Research Objective To develop drugs to treat Calcific Aortic Valve Disease (CAVD), the third leading cause of adult heart disease, by screening a stem cell-based platform based on CAVD patient-derived…
Research Objective Autologous human dopaminergic neurons derived from patient-specific induced pluripotent stem cells Impact Parkinson's disease Major Proposed Activities Characterize differentiation from all 10 patient cell linesCharacterize functionality of patient…
Research Objective Mesenchymal stem cells will be used to deliver an artificial transcription factor to neurons in the brain to treat a genetic disease. Impact It could lead directly to…
Research Objective The goal of this proposal is to develop an optimized, scalable process to manufacture high quality oligodendrocyte precursor cells (OPCs) from human pluripotent stem cells for treating human…
Research Objective Through this project, we would like to evaluate how this T-iPSC-based immunotherapy that we have developed can eliminate lung cancer cells effectively in vivo using xenografted SCID mice.…
Research Objective Gene correction of muscle stem cells Impact These studies will develop a gene editing based therapy for one of the most prevalent lethal childhood disorders called Duchenne Muscular…
Research Objective Demonstrate that our HitFinder™ library can be screened for phenotypic changes in A53T-IPSC-derived dopaminergic neurons and use a secondary handle to identify the targets responsible. Impact This technology…
Research Objective We propose a new approach to differentiation therapy for acute myeloid leukemia by producing local level of high-dose vitamin D in bone marrow via cell therapy with engineered…
Stem cell-based therapy is recognized as a promising therapeutic approach for treating various diseases that are currently intractable. One strategy in regenerative medicine is to transplant stem cells or their…
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