Article
Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing.
Circulation research - 15 Aug 2014
Ding Qiurong, Strong Alanna, Patel Kevin M, Ng Sze-Ling, Gosis Bridget S, Regan Stephanie N, Cowan Chad A, Rader Daniel J, Musunuru Kiran
Abstract excerpt
RATIONALE: Individuals with naturally occurring loss-of-function proprotein convertase subtilisin/kexin type 9 (PCSK9) mutations experience reduced low-density lipoprotein cholesterol levels and protection against cardiovascular disease. OBJECTIVE: The goal of this study was to assess whether gen...
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