Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US6582692B1 · kind B1 · utility
69Cited by
16References
8Claims
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Key dates
| Filing date | Nov 17, 2000 |
| Grant date | Jun 24, 2003 |
| Priority date | — |
| Expiry date | Nov 17, 2020 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2840/44
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
AAV expression vectors and recombinant virions produced using these vectors, which include genes coding for enzymes defective or missing in lysosomal storage disorders, are described. These recombinant AAV virions are useful in the treatment of a variety of lysosomal storage disorders and the methods described herein provide for long-term, sustained expression of the defective or missing enzyme.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.