Methods for increasing the efficiency of recombinant AAV product
US6548286B1 · kind B1 · utility
Assignees
Inventors
Key dates
| Filing date | Apr 14, 1998 |
| Grant date | Apr 15, 2003 |
| Priority date | — |
| Expiry date | Apr 14, 2018 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14152
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention relates to methods and compositions for increasing the production of high titre stocks of recombinant AAV (rAAV) through regulation of expression of the AAV REP and CAP proteins. The methods and compositions of the invention are based on the observation that the low level expression of the AAV REP protein increases the production of AAV viral capsid protein and efficiency of packaging resulting in production of higher titre recombinant viral stocks. The invention encompasses recombinant AAV vectors that direct the expression of AAV REP and CAP proteins and the use of such vectors for the production of novel stable cell lines capable of generating high titre rAAV vectors. The invention provides methods for regulating the expression of the AAV REP gene at the transcriptional and post-translational level. The methods and compositions of the invention can be used to produce high titre stocks of rAAV which can be used in gene therapy for the purpose of transferring genetic information into appropriate host cells for the management and correction of human diseases including inherited and acquired disorders.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.