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.
(Photo Credit: The Stop ALD Foundation)
Amber Salzman, president of the Stop ALD Foundation, comments on Science publication describing gene therapy success for adrenoleukodystrophy (ALD).
Ms. Salzman is a founding member of The Stop ALD Foundation. One of her children, as well as two nephews, were diagnosed with ALD 8 years ago. She discusses the foundation's tireless drive to find a safe treatment option for children diagnosed with this lethal brain disease. Adrenoleukodystrophy was the disease highlighted in the film "Lorenzo's Oil."
(Photo Credit: The Stop ALD Foundation)
Dr. Nathalie Cartier comments on the advantages offered by gene therapy over traditional allogenic stem cell transplant. Her comments follow the successful gene therapy trial for the fatal brain disease adrenoleukodystrophy (ALD) as reported in Science. Dr. Cartier was the study's lead author where two boys were treated with a lentiviral gene therapy.
(Photo Credit: The Stop ALD Foundation)
Source: The StopALD Foundation