Products and methods for treating muscular dystrophy
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsNicolas Sebastien Wein, Kevin Flanigan
Life of this patent
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AssigneeResearch Institute At Nationwide Children's Hospital
InventorsNicolas Sebastien Wein, Kevin Flanigan
Estimating the term…
Products and methods for treating or preventing muscular dystrophies in patients with mutations in any of exons 6, 7, 8, or 9 in their DMD gene are provided. Gene therapy vectors, such as adeno-associated virus (AAV) vectors, and methods of using these vectors to deliver nucleic acids comprising DMD antisense sequences in regulating or restoring expression of transcripts of the DMD gene and a functional form of the dystrophin protein are provided. The products and methods are used for treating, ameliorating and/or preventing muscular dystrophies, such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
A nucleic acid comprising a nucleotide sequence selected from the group consisting of:
Dependencies are inferred from the claim text.