CRISPR Therapeutics AG
GENE-EDITING SYSTEMS FOR MODIFYING A SCN9A OR SCN10A GENE AND METHODS OF USE THEREOF

Abstract:

Disclosed herein are highly efficient gene-editing systems for editing a voltage-gated sodium channel gene, such as sodium voltage-gated channel alpha subunit 9 (SCN9A) or sodium voltage-gated channel alpha subunit 10 (SCN10A), either in vitro or in vivo. The gene-editing systems disclosed herein comprise RNA-guided DNA endonuclease and specific guide RNAs. Also provided herein are uses of the gene-editing systems to modify the target gene, thereby alleviating pain.