NIH R01 · 2024
PROJECT SUMMARY Familial hypercholesterolemia (FH) affects 1 in 250 people and is characterized by impaired low-density lipoprotein (LDL) metabolism resulting in premature cardiovascular disease. CRISPR-Cas9-induced loss of function mutations in the gene encoding Angiopoietin-like 3 (ANGPTL3) has been proposed as a therapeutic strategy to permanently reduce LDL cholesterol and triglyceride levels and lower cardiovascular disease risks. However, the absence of safe and effective methods for delivering CRISPR components into hepatocytes is a major barrier. Adeno-associated viruses (AAVs) are the platform of choice for delivering gene-editing reagents but are associated with severe…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.