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 We propose to discover a tool that will utilize patient specific iPSC-derived human mini-guts to identify personalized antifibrotic treatments in pediatric Crohn’s disease patients Impact The major bottleneck…
Research Objective Development of a screen using inner ear sensory hair cell-like cells made by direct lineage reprogramming, for discovering drugs to ameliorate hearing loss during cancer chemotherapy. Impact Hearing…
Research Objective We will combine a novel method to produce T cells from stem cells with gene editing tools, to create pluripotent stem cells that can serve as a universal…
Research Objective An advanced technology is sought to replace the toxic, ineffective and highly processed components in legacy cryopreservation media. This technology is a fundamentally novel non-toxic freezing media. Impact…
Research Objective This project aims to develop a sensitive and non-invasive method for tracking stem cells in clinical trial, without the need for genetically engineered reporters or long-lived radioisotopes. Impact…
Research Objective To create a universal donor blood cell line that can be used to produce human red blood cells for transplantation. Impact Successful completion of this work would create…
Research Objective The proposed studies will develop three-dimensional cell culture methods for creating human brain neural circuits for disease research and drug discovery. Impact The proposed research will develop a…
Research Objective Use gene editing to create tools for the study of mechanisms by which patient-observed mutations lead to myeloproliferative neoplasms. Impact Editing reagents will yield new insight into how…
Research Objective To increase the efficiency of generating pure retinal progenitor cultures for use in transplantation and to probe general aspects of retinal development. Impact Our methods could increase the…
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