Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Cartier, Nathalie; Hacein-Bey-Abina, Salima; C. Bartholomae, Cynthia; Veres, Gabor; Schmidt, Manfred; Kutschera, Ina; Vidaud, Michel; Abel, Ulrich; Dal-Cortivo, Liliane; Caccavelli, Laure; Mahlaoui, Nizar; Kiermer, Véronique; Mittelstaedt, Denice; Bellesme, Céline; Lahlou, Najiba; Lefrère, François; Blanche, Stéphane; Audit, Muriel; Payen, Emmanuel; Leboulch, Philippe; l’Homme, Bruno; Bougnères, Pierre; Von Kalle, Christof; Fischer, Alain; Cavazzana-Calvo, Marina; Aubourg, Patrick

doi:10.1126/science.1171242

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Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Shared by NobleBlocks on Nov 6, 2009 • 12:00 AM UTC

Authors:

Nathalie Cartier

Salima Hacein-Bey-Abina

Cynthia C. Bartholomae

Abstract

Slowing Brain Disease with Gene Therapy X-linked adrenoleukodystrophy (ALD), the hereditary brain demyelinating disorder that was featured in the movie “ Lorenzo's Oil ,” is typically treated by transplantation of bone marrow from matched donors. This treatment slows progression of the disease by in...

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