Research Objective We will use human patient induced pluripotent stem cell (hiPSC)-based models to screen for a drug that activates a transcription factor critical to the treatment of Autism Spectrum…
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 develop a non-viral gene editing technology to replace the endogenous TCRα locus of stem memory T cells with transgene TCRs that are specific to brain cancer…
Research Objective Universal Pluripotent Liver Failure Therapy (UPLiFT) is composed of 2 lines- UPLiFT0 ( from LiPSC-GR1.1) and UPLiFT1 which will be derived from gene edited universal human pluripotent stem…
Research Objective We will discover small molecule compounds that correct disease in eyecups (retinal organoids) differentiated from patient iPSCs with photoreceptor diseases. Impact Our small molecule agents will provide new…
Research Objective We aim to uncover a therapeutic approach to attempt to treat and potentially cure HIV infection using gene modified blood forming stem cells to enhance the immune response…
Research Objective We will develop a biotherapeutic/monoclonal antibody that blocks the growth of human AML cancer stem cells in vitro and in vivo. Impact Treatment of the cancer stem cell…
Research Objective To develop a consistent and abundant source of transplantable human hepatocytes for transplantation. Impact Developing an abundant and consistent source of human hepatocytes that can be used to…
Research Objective Development of hESC-derived pancreatic beta cells that are protected from allogeneic and autoimmune attack into a cell therapy for type 1 diabetes (T1D) Impact Cell therapy of T1D…
Research Objective Therapeutic candidate to cure hemophilia A is AAV-based genome editing vector that corrects the disease-causing mutation in the factor VIII gene in patient stem cells to develop a…
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