the disease made famous by the film "Lorenzo's Oil" is now being treated experimentally with gene therapy.
The BBC reports:
They used a harmless version of the HIV virus as a vector to carrying a corrected version of the gene into the bone marrow cell.
The HIV virus, which can lead to Aids, is the only one known to be capable of carrying a gene into the nucleus of non-dividing cells... the process needed if the treatment is to have a long-term effect.
The treated bone marrow was then reinjected back into the boys.
Results at both six months and a year suggested the production of the missing enzyme had noticeably improved.
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