Editas Medicine, Inc.

University of Florida Research Foundation Inc

Massachusetts Eye and Ear Infirmary

Morgan Lee Maeder

David A Bumcrot

Shen Shen

Michael Stefanidakis

Rina J Mepani

Ari E Friedland

G Grant Welstead

Sanford Leon Boye

Shannon Elizabeth Boye

Carrie M Margulies

Qin Liu

Eric A Pierce

Deepak Reyon

Joshua C Tycko

Penrose Odonnell

Nachiket D Pendse

Sebastian W Gloskowski

Patrick David Hsu

Nicholas C Huston

Carrie Marie Margulies

CRISPR/CAS-related compositions and methods for treatment of Primary Open Angle Glaucoma (POAG) are disclosed.

Crispr/CAS-related methods and compositions for treating primary open angle glaucoma

Compositions for use in treating subjects with USH2A-associated retinal and/or cochlear degeneration that result from mutations in exon 13 of the USH2A gene by deletion of exon 13 from the USH2A gene or transcripts, and methods of use thereof, as well as genetically modified animals and cells.

CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated usher syndrome

CRISPR/CAS-related compositions and methods for treatment of Leber's Congenital Amaurosis 10 (LCA10) are disclosed.

CRISPR/Cas-related methods and compositions for treating Leber's congenital amaurosis 10 (LCA10)

Provided herein are methods and compositions for treating an eye disorder, for example cone-rod dystrophy type 6 (CORD6). In certain aspects, a therapeutically effective amount of a composition comprising nucleic acids is administered to a subject to treat an autosomal dominant disorder or condition, such as a condition associated with a dominant mutation in a guanylate cyclase 2D (GUCY2D) gene, such as knocking out a dominant mutant form of the gene in the subject. Further provided herein are recombinant AAV particles that comprise one or more recombinant AAV genomes comprising nucleic acids that encode a guide RNA that targets a GUCY2D gene and/or an RNA-guided endonuclease.

Methods and compositions for treating cone-rod retinal dystrophy

Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease.

Compositions and methods for treating CEP290 associated disease

Compositions and methods for treatment of CEP290 related diseases are disclosed.

Compositions and methods for treating CEP290-associated disease

CRISPR/CAS-related methods and compositions for treating Leber's Congenital Amaurosis 10 (LCA10)

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

CRISPR/Cas-related compositions and methods for treatment of Usher Syndrome and/or Retinitis Pigmentosa are disclosed herein.

Growing community of inventors

Jamaica Plain, MA, United States of America

Morgan Lee Maeder