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EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY

  • US 20140329881A1
  • Filed: 03/14/2014
  • Published: 11/06/2014
  • Est. Priority Date: 03/14/2013
  • Status: Abandoned Application
First Claim
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1. An antisense oligomer of 20-50 nucleotides in length capable of binding a selected target to induce exon skipping in the human dystrophin gene, wherein said antisense oligomer comprises a sequence of bases that specifically hybridizes to an exon 44 target region selected from the group consisting of H44A(−

  • 07+15), H44A(−

    08+15), H44A(−

    06+15), H44A(−

    08+17), H44A(−

    07+17), and H44A(−

    06+17), wherein the bases of said oligomer are linked to morpholino ring structures, and wherein said morpholino ring structures are joined by phosphorous-containing intersubunit linkages joining a morpholino nitrogen of one ring structure to a 5′

    exocyclic carbon of an adjacent ring structure.

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