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

Cartier, Nathalie; Hacein‐Bey‐Abina, Salima; C. Bartholomae, Cynthia; Veres, Gábor; 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, Marina; Aubourg, Patrick

doi:10.1126/science.1171242

Public

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

Published in Science • Nov 5, 2009

Authors:

Nathalie Cartier

Salima Hacein‐Bey‐Abina

Cynthia C. Bartholomae

Abstract

X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that is caused by a deficiency in ALD protein, an adenosine triphosphate-binding cassette transporter encoded by the ABCD1 gene. ALD progression can be halted by allogeneic hematopoietic cell transplantation (HCT). W...

Subject

Adrenoleukodystrophy

Genetic enhancement

Haematopoiesis

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