Compositions and methods for treating spinal muscular atrophy
US10821154B2 · kind B2 · utility
4Cited by
14References
19Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | May 1, 2013 |
| Grant date | Nov 3, 2020 |
| Priority date | — |
| Expiry date | Jul 5, 2033 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14171
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN. In one aspect, the viral particles are administered the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.