Why the study?
Does in vivo AAV-CRISPR/Cas9-mediated Ldlr gene correction improve LDLR expression and ameliorate atherosclerosis phenotypes in Ldlr mutants?
Does in vivo AAV-CRISPR/Cas9-mediated Ldlr gene correction improve LDLR expression and ameliorate atherosclerosis phenotypes in Ldlr mutants?
AAV-CRISPR/Cas9-mediated Ldlr gene correction ameliorates atherosclerosis in a preclinical model, suggesting a potential therapeutic approach for familial hypercholesterolemia.
Hypothesis-generating for CRISPR gene editing in FH; leaves open human translation, efficacy, and safety.
Our work shows that in vivo AAV-CRISPR/Cas9-mediated Ldlr gene correction can partially rescue LDLR expression and effectively ameliorate atherosclerosis phenotypes in Ldlr mutants, providing a potential therapeutic approach for the treatment of patients with familial hypercholesterolemia.
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Zhao et al. (2019) studied this question.
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