Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping

非人类灵长类动物接受临床相关剂量的 AAV9.U7snRNA 载体（旨在诱导 DMD 外显子 2 跳跃）后未出现毒性