Madelynn N Whittaker1,2, Lauren C Testa1, Aidan Quigley1

  • 1Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA.

概括

使用CRISPRoff进行表观基因组编辑,通过甲基化实现了PCSK9基因表达的持久淘汰,提供了潜在的心血管疾病治疗方法. 然而,耐久性在目标基因之间有所不同,限制了更广泛的治疗应用.

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