Dr. Patrick Aubourg Discusses the ALD Gene Therapy Trial (VIDEO)

The StopALD Foundation

Caption

Dr. Patrick Aubourg comments on the successful gene therapy trial for the fatal brain disease adrenoleukodystrophy (ALD) as reported in Science. Dr. Aubourg was the leading investigator on this study, and treated two boys with a lentiviral gene therapy. Without treatment these boys would otherwise have faced a fatal prognosis.

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The Stop ALD Foundation

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