Antisense oligomer compounds

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.

1 . 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) 
     
     
         5 . The modified antisense oligonucleotide or pharmaceutically acceptable salt thereof of  claim 1 , wherein the modified antisense oligonucleotide is conjugated to a peptide. 
     
     
         6 - 18 . (canceled) 
     
     
         19 . The modified antisense oligonucleotide or pharmaceutically acceptable salt thereof of  claim 1 , wherein the modified antisense oligonucleotide or pharmaceutically acceptable salt thereof promotes skipping of exon 44 in the processing of a human dystrophin pre-processed mRNA. 
     
     
         20 . A pharmaceutical composition comprising the modified antisense oligonucleotide or pharmaceutically acceptable salt thereof of  claim 1 , and a pharmaceutically acceptable carrier. 
     
     
         21 . A method for treating a muscular dystrophy in a patient in need thereof comprising administering to the patient the modified antisense oligonucleotide or pharmaceutically acceptable salt thereof of  claim 1 .