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COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY

  • US 20100016215A1
  • Filed: 06/26/2009
  • Published: 01/21/2010
  • Est. Priority Date: 06/29/2007
  • Status: Abandoned Application
First Claim
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1. An antisense compound for use in treating myotonic dystrophy DM1 or DM2, comprisingan antisense oligonucleotide having 8-30 bases, with at least 8 contiguous bases being complementary to the polyCUG or polyCCUG repeats in the 3′

  • UTR region of dystrophia myotonica protein kinase (DMPK) mRNA in DM1 or DM2, respectively, andconjugated to the oligonucleotide, a cell-penetrating peptide having the sequence (RXRR(B/X)R)2XB, where R is arginine;

    B is β

    -alanine; and

    each X is —

    C(O)—

    (CH2)n

    NH—

    , where n is 4-6.where the compound is effective to selectively block the sequestration of at least one of muscleblind-like 1 protein (MBNL1) and CUGBP in heart and quadricep muscle in a myotonic dystrophy animal model.

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