Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies
US8058061B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Apr 30, 2009 |
| Grant date | Nov 15, 2011 |
| Priority date | — |
| Expiry date | Sep 24, 2029 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2740/15043
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Recombinant lentiviral vectors having a region encoding a functional β-globin gene; and large portions of the β-globin locus control regions which include DNase I hypersensitive sites HS2, HS3 and HS4 provides expression of β-globin when introduced into a mammal, for example a human, in vivo. Optionally, the vector further includes a region encoding a dihydrofolate reductase. The vector may be used in treatment of hemoglobinopathies, including β-thalessemia and sickle-cell disease. For example, hematopoietic progenitor or stem cells may be transformed ex vivo and then restored to the patient. Selection processes may be used to increase the percentage of transformed cells in the returned population. For example, a selection marker which makes transformed cells more drug resistant than untransformed cells allows selection by treatment of the cells with the corresponding drug.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.