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Methods: The aim of this study was to determine whether in vivo somatic cell gene editing by adeno-associated virus within the CRISPR/Cas9 device delivered by adeno-associated virus could cure familial hypercholesterolemia in a mouse model. The AAV-CRISPR/Cas9 was intended to correct a point mutation in the Ldlr gene in hepatocytes and was delivered subcutaneously to Ldlr E208X mice. After a high-fat diet regimen and the Ldlr mutation was corrected in a subset of hepatocytes after AAV-CRISPR/Cas9 therapy, with LDLR protein expression partially restored. Conclusions: In vitro AAV-CRISPR/Cas9 gene mutation can partially restore LDLR expression and effectively eliminate atherosclerosis phenotypes in Ldlr mutants, providing a potential therapeutic solution for patients with familial hypercholesterolemia.
Source link: https://doi.org/10.1161/circulationaha.119.042476
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