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Gene Therapy for Adrenoleukodystrophy

This article discusses a new Science paper (6 November 2009: Vol. 326. no. 5954, pp. 818 – 823 DOI: 10.1126/science.1171242) about gene therapy for a brain disease that affects myelination in the brain.  The method used was similar to previous gene therapy trials which is to destroy the cells that produce stem cells and then infuse the body with genetically modified white blood cells.  The genetically modified white blood cells would then secrete myelin.  The two patients in this trial both exhibited dramatic reduction in brain lesions.  A novel aspect of this paper, aside from the treated disease, is the use of an HIV derived vector which has higher efficiency in infection that previously used retroviral vectors.  The vector was also designed to self-inactivate so as not to produce any RNA making it safer than previous vectors.

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Posted in Exciting Advances, Gene Therapy, Science News Links.

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