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US 2026/0258419 A1Published application
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
Life of this patent
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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 rAAV in the treatment of Duchenne Muscular Dystrophy.
Claims19
A nucleic acid comprising at least one nucleotide sequence or a combination of two or more exon 2-targeted nucleotide sequences, wherein each nucleotide sequence is set forth in any one of SEQ ID NOs:
- 4, 5, 7 and 8.
Dependencies are inferred from the claim text.