Sarepta Therapeutics, Inc.

Jason Gatlin

Patrick L Iversen

Frederick Joseph Schnell

Baozhong Cai

A modified antisense oligonucleotide of about 10 to about 40 nucleobases is disclosed. The oligonucleotide comprises a targeting sequence having a region complementary to at least one string of three or more identical contiguous nucleobases in a target sequence, wherein the target sequence comprises at least one additional nucleobase compared to the region of the targeting sequence and the at least one additional nucleobase has no complementary nucleobase in the region of the targeting sequence, and wherein the targeting region complementary to the at least one string of three or more identical contiguous nucleobases is internal to the targeting sequence.

Growing community of inventors

Cambridge, MA, United States of America

Jason Gatlin