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US 10,471,089 B2Granted
Combined therapy for duchenne muscular dystrophy
AssigneesAssociation Institut De Myologie, Sorbonne Universite, Centre National De La Recherche Scientifique, Inserm (Institut National De La Sante Et De La Recherche Medicale)
InventorsStephanie Lorain, Thomas Voit, Matthew Wood, Graham McClorey
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Abstract
The present invention relates to the combined use of antisense oligonucleotides and viral vectors for the treatment of Duchenne muscular dystrophy.
Claims13
A combination therapy method comprising:
- administering to a human subject an isolated antisense oligonucleotide (AON) between 10 and 40 nucleotides in length capable of inducing an exon-skipping in a dystrophin pre-mRNA thereby inducing functional dystrophin expression, and
- subsequently administering to the subject at least one viral vector encoding a Duchenne muscular dystrophy therapeutic product selected from (i) an antisense oligonucleotide able to induce exon-skipping within a dystrophin pre-mRNA, (ii) a dystrophin gene-editing endonuclease, and (iii) a functional dystrophin protein;
- wherein the pretreatment with the AON prevents the loss of therapeutic viral vector genomes from the muscles of the subject.
Dependencies are inferred from the claim text.