X-linked adrenoleukodystrophy gene and corresponding protein

Details X-linked adrenoleukodystrophy gene and corresponding protein X-linked adrenoleukodystrophy gene and corresponding protein

1995-06-07

1999-02-09

Campell, Bruce R.

Drug, bio-affecting and body treating compositions

Whole live micro-organism, cell, or virus containing

Genetically modified micro-organism, cell, or virus

4351723, 4353201, 435375, 935 62, A01N 6300, A61K 4800

Patent

active

058690395

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.

REFERENCES:
patent: 5227170 (1993-07-01), Sullivan
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