Vertex Pharmaceuticals Incorporated
GENE-EDITING SYSTEMS FOR EDITING A CYSTIC FIBROSIS TRANSMEMBRANE REGULATOR (CFTR) GENE

Abstract:

Described herein are highly efficient gene-editing systems comprising a nuclease, a guide RNA, and/or a donor template and uses thereof for editing a cystic fibrosis transmembrane regulator (CFTR) gene either in vitro or in vivo.