Patrick Aubourg

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

Nathalie Cartier, Salima Hacein‐Bey‐Abina, Cynthia C. Bartholomae et al. · Science · 2009 · 1.5K citations · Full text

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Marina Cavazzana, Emmanuel Payen, Olivier Nègre et al. · Nature · 2010 · 1.3K citations

Hmga2 Activation, Transfusion Independence, Genetics +7

Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters

Jean Mosser, Anne‐Marie Douar, Claude‐Olivier Sarde et al. · Nature · 1993 · 1.2K citations

Molecular Physiology, Signal Transduction, Abc Transporters +4

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Florian Eichler, Christine Duncan, Patricia L. Musolino et al. · New England Journal of Medicine · 2017 · 575 citations · Full text

X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management

Marc Engelen, Stephan Kemp, Björn M. van Geel et al. · Orphanet Journal of Rare Diseases · 2012 · 555 citations · Full text