The General Hospital Corporation

Editas Medicine, Inc.

Deepak Reyon

David A Bumcrot

Bradley Bernstein

Morgan Lee Maeder

Ari E Friedland

G Grant Welstead

Jae Keith Joung

Eric M Mendenhall

Bradley E Bernstein

Fusion proteins comprising a DNA binding domain, e.g., a TAL effector repeat array (TALE) or zinc finger array, and a catalytic domain comprising a sequence that catalyzes histone demethylation, and methods of use thereof.

Transcription activator-like effector (tale) - lysine-specific demethylase 1 (LSD1) fusion proteins

Disclosed herein are genome editing systems and compositions that target a cystic fibrosis transmembrane conductance regulator (CFTR) gene and a sodium channel epithelial 1 alpha (SCNN1A) gene, comprising a Cas9 molecule, and a gRNA molecule comprising a targeting domain that is complementary with a target sequence of a CFTR gene or a SCNN1A gene, and cells comprising such genome editing systems and compositions. Also provided are methods for using the genome editing systems, compositions, and cells for genome engineering (e.g., altering a CFTR gene and/or a SCNN1A gene), and for preventing or treating Cystic Fibrosis (CF) and CF-like disease.

CRISPR/CAS-related methods and compositions for treating cystic fibrosis

Growing community of inventors

Malden, MA, United States of America

Deepak Reyon