In-vivo CRISPR/Base-Editing “One-Shot” Therapies Targeting PCSK9
The background of PCSK9 as a target, the gene editing machinery and the preliminary results are described, and the unique safety challenges of this technology are explored and considerations for appropriate patient populations are proposed to maintain net positive benefits despite unknowns.
Benjamin R. Nelson, A. Khera
· Current Atherosclerosis Repo... · 0 citations