Compositions and methods for the expression of factor VIII polypeptides and uses therefor
US6517830B1 · kind B1 · utility
8Cited by
4References
12Claims
0Family size
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Key dates
| Filing date | Aug 4, 2000 |
| Grant date | Feb 11, 2003 |
| Priority date | — |
| Expiry date | Aug 4, 2020 |
Classification
- Technology area (CPC A)Human Necessities
- CPC primaryA61K48/00
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Compositions and methods are provided for the in vivo gene delivery of nucleic acid sequences encoding the factor VIII protein to the liver endothelial sinusoidal cells (LSECs). Compositions and methods are also provided for the ex vivo gene transfer of nucleic acid sequences encoding the factor VIII protein to cultured LSECs and the implantation of the transformed LSECs in vivo. These methods and compositions increase the level of factor VIII in the blood stream and find use in the gene therapy treatment of hemophilia A.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.