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1. (WO2018165504) SUPPRESSION OF PAIN BY GENE EDITING

Pub. No.:    WO/2018/165504    International Application No.:    PCT/US2018/021664
Publication Date: Fri Sep 14 01:59:59 CEST 2018 International Filing Date: Sat Mar 10 00:59:59 CET 2018
IPC: C12N 15/113
C12N 9/16
C12N 9/22
Applicants: PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Inventors: MAIANTI, Juan, Pablo
LIU, David, R.
Title: SUPPRESSION OF PAIN BY GENE EDITING
Abstract:
Provided herein are systems, compositions, kits, and methods for the suppression of pain (e.g., chronic pain). Genes encoding ion channels (e.g., SCN9A) responsible for the propagation pain signals in neurons (e.g., DRG neurons) may be edited using a genome editing agent (e.g., a nucleobase editor). In some embodiments, loss-of-function ion channel mutants are generated, leading to pain suppression. In some embodiments, the genome editing agent is administered locally to the site of pain or to the nerves responsible for propagation of the pain signal.