#CRISPR paper: Disruption of PCSK9 in liver lowers cholesterol in mice.

PCSK9 is an attractive target as inhibitors reduce serum cholesterol and inactivating mutations are associated with reduced cholesterol as well; conversely gain-of-function mutations are associated with familial hypercholesterolemia.  Knockdown approaches in adult mouse liver had been done previously for PCSK9 but not yet with CRISPR.

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

Qiurong Ding, 

Alanna Strong, 

Kevin M. Patel, 

Sze-Ling Ng,

Bridget S. Gosis, 

Stephanie N. Regan, 

Chad A. Cowan, 

Daniel J. Rader, 

Kiran Musunuru

.   Circ Res. 2014 Aug 15;115(5):488-92. doi: 10.1161/CIRCRESAHA.115.304351. Epub 2014 Jun 10.