The National Heart, Lung, and Blood Institute (NHLBI) awarded a $667,704 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837) to The Trustees of the University of Pennsylvania, doing business as Clinical Practices of the University of Pennsylvania. The funding supports research to develop chemically inducible split base editors that provide precise and controllable in vivo genome editing for the treatment of hypertrophic cardiomyopathy (HCM). The research aims to expand chemically-induced dimerization systems, optimize the splitting strategy, and create variants of split base editors to achieve versatile base editing with low off-target activity. The project seeks to validate these split base editor systems by correcting MYBPC3-associated HCM and decreasing PCSK9 expression and blood cholesterol levels in vivo. The award is effective from Feb 1, 2025 through Nov 30, 2028.
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