Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
US8648053B2 · kind B2 · utility
2Cited by
1References
21Claims
0Family size
Assignee
Inventor
Key dates
| Filing date | May 1, 2012 |
| Grant date | Feb 11, 2014 |
| Priority date | — |
| Expiry date | May 1, 2032 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/33
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention provides a method for treating Usher's syndrome in a human subject including administering to the human subject an oligonucleotide having 8 to 30 linked nucleosides having a nucleobase sequence comprising a complementary region comprising at least 8 contiguous nucleobases complementary to a target region of equal length within exon 3 of an Usher RNA transcript.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.