Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Eichler, Florian; Duncan, Christine; L. Musolino, Patricia; J. Orchard, Paul; De Oliveira, Satiro; J. Thrasher, Adrian; Armant, Myriam; Dansereau, Colleen; C. Lund, Troy; P. Miller, Weston; V. Raymond, Gerald; Sankar, Raman; J. Shah, Ami; Sevin, Caroline; Bobby Gaspar, H.; Gissen, Paul; Amartino, Hernán; Bratkovic, Drago; Smith, Nicholas; Paker, Asif; Shamir, Esther; O’Meara, Tara; Davidson, David; Aubourg, Patrick; A. Williams, David

doi:10.1056/nejmoa1700554

Public

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Published in New England Journal of Medicine • Oct 4, 2017

Authors:

Florian Eichler

Christine Duncan

Patricia L. Musolino

Abstract

BACKGROUND: In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and death, can be halted only with allo...

Subject

Adrenoleukodystrophy

Medicine

Hematopoietic stem cell transplantation

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