Methods and compositions relating to improved lentiviral vector production systems
US8900858B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Oct 13, 2009 |
| Grant date | Dec 2, 2014 |
| Priority date | — |
| Expiry date | Dec 4, 2029 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2840/44
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention provides HIV-derived lentivectors which are multiply modified to create highly safe, efficient, and potent vectors for expressing transgenes for gene therapy. The lentiviral vectors comprise various combinations of an inactive central polypurine tract, a stuffer sequence, which may encode drug susceptibility genes, and a mutated hairpin in the 5′ leader sequence that substantially abolishes replication. These elements are provided in conjunction with other features of lentiviral vectors, such as a self-inactivating configuration for biosaftey and promoters such as the EF1α promoter as one example. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element. These vectors therefore provide useful tools for genetic treatments for inherited and acquired disorders, gene-therapies for cancers and other disease, the creation of industrial and experimental production systems utilizing transformed cells, as well as for the study of basic cellular and genetic processes.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.