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Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs

  • US 9,862,945 B2
  • Filed: 04/18/2014
  • Issued: 01/09/2018
  • 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 recombinant AAV comprises the genome insert set out in SEQ ID NO:

  • 2.

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