Effective gene therapy tools for dystrophin exon 53 skipping
US10752898B2 · kind B2 · utility
31Cited by
2References
15Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Dec 9, 2016 |
| Grant date | Aug 25, 2020 |
| Priority date | — |
| Expiry date | Mar 21, 2037 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/33
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The invention relates to a recombinant adeno-associated viral vector (rAAV) comprising a sequence encoding an antisense oligonucleotide (AON) directed against a segment of at least 33 bases from the +30 to +69 region of exon 53 of the pre-messenger RNA (pre-mRNA) of dystrophin, advantageously of human origin, and to the use thereof as a drug, in particular for the treatment of Duchenne muscular dystrophy (DMD).
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.