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SMN2 element 1 antisense compositions and methods and uses thereof

  • US 9,885,040 B2
  • Filed: 04/11/2014
  • Issued: 02/06/2018
  • Est. Priority Date: 04/12/2013
  • Status: Active Grant
First Claim
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1. A composition for blocking the repressive activity of the Element 1 of the SMN2 pre-mRNA, the composition comprising an antisense oligonucleotide that comprises the nucleic acid sequence SEQ ID NO:

  • 17 (v1.11) or the nucleic acid sequence SEQ ID NO;

    17 (v1.11) except for consisting of one nucleotide substitution,wherein the antisense oligonucleotide comprises a Morpholino backbone.

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