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.
Full Text
What is claimed is:
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.
Timeline
Filed
05/15/2026Published
09/03/2026Granted
Not AvailableIPC Codes(7)
C12N 15/113:Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 48/00:Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/47:from mammals