Antisense oligomer compounds
AssigneeSarepta Therapeutics, Inc.
InventorsFrederick J. SCHNELL, Baozhong CAI, Jason GATLIN, Patrick L. IVERSEN
Life of this patent
Estimating the term…
Abstract
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.
Claims7
A modified antisense oligonucleotide or a pharmaceutically acceptable salt thereof comprising a nucleic acid sequence consisting of any one of SEQ ID NOs. 2-7, wherein the modified antisense oligonucleotide contains one or more morpholino groups.
2-4. (canceled)
6-18. (canceled)
Dependencies are inferred from the claim text.