Nathalie Cartier

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

Medicine, Neurology, Neuroscience +17

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

Medicine, Hematology, Molecular Biology +12

A Two-Year Trial of Oleic and Erucic Acids (“Lorenzo's Oil”) as Treatment for Adrenomyeloneuropathy

Patrick Aubourg, C. Adamsbaum, M. C. Lavallard-Rousseau et al. · New England Journal of Medicine · 1993 · 241 citations · Full text

Life Sciences, Neurology, Pharmacotherapy +17

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

Alessandra Biffi, Cynthia C. Bartolomae, Daniela Cesana et al. · Blood · 2011 · 241 citations · Full text

Life Sciences, Pharmaceutical Biotechnology, Gene Therapy +11

Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia

Morgane Perdomini, Brahim Belbellaa, Laurent Monassier et al. · Nature Medicine · 2014 · 230 citations · Full text

Life Sciences, Genetics, Molecular Biology +16