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…

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Development of a low-cost, clinical-grade iPS maintenance medium for enabling stem cell therapy manufacturing

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Translational Candidate HiDef-B8, an iPS cell maintenance medium designed to reduce stem cell GMP manufacturing costs and risk. Area of Impact HiDef-B8 addresses scale-up manufacturing, by being lower-cost ($300/L) and…

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