A new precision medicine based iPSC-derived model to study personalized intestinal fibrosis treatments in pediatric patients with Crohn’s disease

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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…

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A screen for drugs to protect against chemotherapy-induced hearing loss, using sensory hair cells derived by direct lineage reprogramming from hiPSCs

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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…

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Genome editing for causation and reversion of MPN-associated mutations in human hematopoietic stem cells

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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…

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Microenvironment based optimization of retinal induction using CRISPR-CAS9 reporter pluripotent stem cells as an expandable source of retinal progenitors and photoreceptors.

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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…

Continue ReadingMicroenvironment based optimization of retinal induction using CRISPR-CAS9 reporter pluripotent stem cells as an expandable source of retinal progenitors and photoreceptors.