Exon skipping therapy for dysferlinopathies
US8586560B2 · kind B2 · utility
Assignees
Inventors
Key dates
| Filing date | Oct 18, 2010 |
| Grant date | Nov 19, 2013 |
| Priority date | — |
| Expiry date | Oct 18, 2030 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/33
- WIPO fieldBiotechnology
- WIPO sectorChemistry
Abstract
The present invention relates to methods for restoring the function of a mutated dysferlin comprising the step of preventing splicing of one or more exons which encode amino acid sequences that cause said dysferlin dysfunction. Particularly, the splicing of exon 32 is prevented. The present invention also relates to a method for treating a dysferlinopathy in a patient in need thereof, comprising the step of administering to said patient antisense oligonucleotides complementary to nucleic acid sequences that are necessary for correct splicing of one or more exons which encode amino acid sequences that cause said dysfunction. Particularly, the splicing of exon 32 is prevented.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.