The National Heart, Lung, and Blood Institute (NHLBI) awarded Clemson University's Division of Research a $1,123,828 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837). The grant supports research to develop non-viral gene editing delivery methods for treating familial hypercholesterolemia (FH), a genetic disorder characterized by high cholesterol and elevated cardiovascular disease risk. The project will directly compare the efficacy and specificity of CRISPR-Cas9 gene editing delivered via lipid nanoparticles, electroporation, and adeno-associated viruses in primary mouse hepatocytes. The research aims to optimize methods for transplanting gene-edited hepatocytes and using acetaminophen to selectively expand the edited cells in an FH mouse model. As a sub-award, Clemson is collaborating with the Research Foundation for the State University of New York to leverage their expertise in lipid nanoparticle synthesis and formulation for the gene editing applications. The overall project is intended to advance non-viral approaches for delivering CRISPR-based gene editing therapies as a treatment for FH.
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