New patients wanted for 'Lorenzo's oil' gene therapy.

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  • Source:
    New Scientist. 11/14/2009, Vol. 204 Issue 2734, p6-6. 1p.
  • Additional Information
    • Subject Terms:
      RESEARCH; GENE therapy; X-linked intellectual disabilities; BRAIN diseases; MYELIN sheath diseases; GENETIC disorders
    • Abstract:
      The article discusses research on the positive response of two boys to a gene therapy for the brain disease X-linked adrenoleukodystrophy (ALD). It references a study by Nathalie Cartier and colleagues, published in a 2009 issue of the journal "Science." The reseachers collected blood stem cells from two 7-year old boys with ALD, introduced a virus carrying a correct copy of the defective gene into the cells, then re-injected the stem cells. ALD is caused by a defective gene that affects the myelin sheath coating nerves in the brain to wear away.