Methods for delivering DNA to muscle cells using recombinant adeno-associated virus virions to treat lysosomal storage disease
US6335011B1 · kind B1 · utility
52Cited by
5References
13Claims
0Family size
Assignees
Inventors
Key dates
| Filing date | Sep 28, 1999 |
| Grant date | Jan 1, 2002 |
| Priority date | — |
| Expiry date | Sep 28, 2019 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2840/44
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.