Editas Medicine, Inc.

Rina J Mepani

Morgan Lee Maeder

Michael Stefanidakis

David A Bumcrot

Shen Shen

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.

Growing community of inventors

Belmont, CA, United States of America

Rina J Mepani