Gene therapy for treating mucopolysaccharidosis type I
US10973929B2 · kind B2 · utility
0Cited by
16References
25Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Feb 2, 2017 |
| Grant date | Apr 13, 2021 |
| Priority date | — |
| Expiry date | Apr 27, 2037 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2830/15
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
A suspension useful for AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating MPSI and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.