Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsKevin Flanigan, Adeline Vulin-Chaffiol, Nicolas Wein
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Abstract
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
Claims17
A recombinant adeno-associated virus (rAAV) comprising a genome comprising at least one Duchenne Muscular Dystrophy (DMD) exon 2-targeted U7snRNA polynucleotide construct, wherein the at least one DMD exon 2-targeted U7snRNA polynucleotide construct comprises the nucleotide sequence of any one of SEQ ID NOs:
- 4, 5, 7 and 8.
Dependencies are inferred from the claim text.