Gene Therapy for Adrenoleukodystrophy
December 5, 2017 | Terry Sharrer
You may remember Lorenzo Odono and the 1992 movie, “Lorenzo’s Oil,” about his and his family’s struggle with a rare but fatal inherited disease, adrenoleukodystrophy (ALD). This piece describes a follow up story, which also would make a touching movie about an aunt who pursues a gene therapy clinical trial for ALD to treat her nephew. The experiment in France involved inserting the ALD gene into an altered HIV vector which then infected the patient’s bone marrow stem cells, normalizing them. The therapy was curative, but also notice the remark about the treatment’s cost which was researched and developed through publicly funded grants. MORE
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