Whitehead Institute for Biomedical Research

Editas Medicine, Inc.

G Grant Welstead

Rudolf Jaenisch

Alexander Meissner

Ruth Foreman

Yaqub Hanna

Marius Wernig

Christopher J Lengner

Oliver Tobias Brambrink

David A Bumcrot

Morgan Lee Maeder

Ari E Friedland

Jacob Hanna

Deepak Reyon

Rudolph Jaenisch

CRISPR/CAS-related compositions and methods for treatment of Sickle Cell Disease (SCD) are disclosed.

CRISPR/CAS-related methods and compositions for treating sickle cell disease

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

The disclosure relates to a method of reprogramming one or more somatic cells, e.g., partially differentiated or fully/terminally differentiated somatic cells, to a less differentiated state, e.g., a pluripotent or multipotent state. In further embodiments the invention also relates to reprogrammed somatic cells produced by methods of the invention, to uses of said cells, and to methods for identifying agents useful for reprogramming somatic cells.

Reprogramming of somatic cells

Growing community of inventors

Cambridge, MA, United States of America

G Grant Welstead