Antisense compounds targeting genes associated with usher syndrome
US9353371B2 · kind B2 · utility
1Cited by
43References
15Claims
0Family size
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Key dates
| Filing date | May 2, 2012 |
| Grant date | May 31, 2016 |
| Priority date | — |
| Expiry date | May 2, 2032 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/33
- WIPO fieldOrganic fine chemistry
- WIPO sectorChemistry
Abstract
The present invention provides compounds comprising oligonucleotides complementary to an Usher transcript. Certain such compounds are useful for hybridizing to an Usher transcript, including but not limited, to an Usher transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the Usher transcript. In certain such embodiments, the Usher transcript includes a mutation that results in cryptic splicing and hybridization of the oligonucleotide results in a decrease in the amount of cryptic splicing. In certain embodiments, such compounds are used to treat Usher Syndrome.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.