Methods for treating spinal muscular atrophy
US8633019B2 · kind B2 · utility
33Cited by
0References
7Claims
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Key dates
| Filing date | May 27, 2009 |
| Grant date | Jan 21, 2014 |
| Priority date | — |
| Expiry date | Jun 11, 2029 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Q2600/158
- WIPO fieldOrganic fine chemistry
- WIPO sectorChemistry
Abstract
The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.