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ShareWASHINGTON: Scientists are reporting a new breakthrough in the ongoing battle against a fatal brain disease called adrenoleukodystrophy using gene therapy.
The brain condition is hereditary and is characterized by the slow wearing down of fatty acids, which are meant to protect nerves in the brain.
Over time as the condition worsens, the patient becomes more physically and mentally challenged until they eventually die.
The condition primarily strikes young males, and up until now was only treated by administering bone marrow transplants.
In a new report, doctors detail the cases of two boys from France with adrenoleukodystrophy who underwent gene therapy.
Genetically defective bone marrow blood cells were removed from the boys and "corrected" by insertion of properly functioning genes. The treated cells were injected back into the patients.
The therapy halted the progression of ALD, and the boys' conditions remain stable more than two years later. No adverse effects of the gene therapy have been seen by the doctors.
The report is published in the Nov. 6 issue of Science.
