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RECOMBINANT ADENO-ASSOCIATED VIRUS DELIVERY OF EXON 2-TARGETED U7SNRNA POLYNUCLEOTIDE CONSTRUCTS

  • US 20160076028A1
  • Filed: 04/18/2014
  • Published: 03/17/2016
  • Est. Priority Date: 04/20/2013
  • Status: Active Grant
First Claim
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1. A method of ameliorating Duchenne Muscular Dystrophy in a patient with DMD exon 2 duplications in need thereof comprising the step of administering a recombinant adeno-associated virus (rAAV) to the patient, wherein the genome of the rAAV comprises at least one exon 2-targeted U7snRNA polynucleotide construct.

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