X-linked adrenoleukodystrophy gene and corresponding protein
US5869039A · kind A · utility
Assignee
Inventors
Key dates
| Filing date | Jun 7, 1995 |
| Grant date | Feb 9, 1999 |
| Priority date | — |
| Expiry date | Jun 7, 2015 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2799/027
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
A method for the treatment of adrenoleukodystrophy or adrenomyeloneuropathy, in which there is administered to a human patient in need of the same an effective amount of hematopoietic cells modified ex vivo by infection with a retroviral vector containing a nucleic acid fragment comprising a sequence as represented on FIG. 2 (SEQ. ID No: 1) or FIG. 6 (SEQ. ID. No: 4-17) or FIG. 7 (SEQ. ID No: 18-23). Also, human hematopoietic cells can be treated ex vivo to correct the content of very long chain fatty acids in them, by providing human hematopoietic cells of a patient affected by adrenoleukodystrophy or adrenomyeloneuropathy, and infecting them ex vivo with a retroviral vector containing, operably linked to a promoter, a nucleic acid fragment as above. The expression of the nucleic acid in the hematopoietic cells corrects the content of very long chain fatty acids in the hematopoietic cells.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.