LDLR and familial hypercholesterolemia: In an animal model researchers indicated that adeno-associated virus (AAV) serotype 9-based delivery of the Cas9 system can efficiently edit cardiomyocytes through specifically targeting the disease-causing allele.36 Interestingly, other in vivo model indicated that AAV-CRISPR/Cas9–mediated Ldlr gene correction can ameliorate atherosclerosis phenotypes and be a potent treatment strategy for patients with familial hypercholesterolemia.37