Modified RNA-Based Gene Therapy for Cardiac Regeneration Through Cardiomyocyte Proliferation

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Research Objective Efficacious and safe intramyocardial delivery of modified mRNA encoding cell cycle regulators as a gene therapy for cardiac regeneration through resident cardiomyocyte proliferation. Impact This project would provide…

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Developing gene therapy for dominant optic atrophy using human pluripotent stem cell-derived retinal organoid disease models

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Research Objective We will develop a gene therapy for a major inherited optic nerve disease and test the effectiveness of the treatment by analyzing healthy and patient stem cell-derived mini…

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