Patrick Aubourg

Overview

Best Publications

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

  Nathalie Cartier;Salima Hacein-Bey-Abina;Cynthia C. Bartholomae;Gabor Veres

  1787
  Citations

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

  Marina Cavazzana-Calvo;Emmanuel Payen;Olivier Negre;Gary Wang

  1620
  Citations

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

  Jean Mosser;Anne-Marie Douar;Claude-Olivier Sarde;Petra Kioschis

  1435
  Citations

• Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

  Florian Eichler;Christine Duncan;Patricia L. Musolino;Paul J. Orchard

  605
  Citations

• Senataxin, the ortholog of a yeast RNA helicase, is mutant in ataxia-ocular apraxia 2

  M C Moreira;S Klur;M Watanabe;A H Nemeth

  604
  Citations

• Efficient derivation of microglia-like cells from human pluripotent stem cells

  Julien Muffat;Yun Li;Bingbing Yuan;Maisam Mitalipova

  600
  Citations

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

  Marc Engelen;Stephan Kemp;Marianne de Visser;Björn M van Geel

  536
  Citations

• Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation.

  Patrick Aubourg;Stéphane Blanche;Isabelle Jambaqué;Francis Rocchiccioli

  457
  Citations

• Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999.

  Charles Peters;Lawrence R. Charnas;Ye Tan;Richard S. Ziegler

  408
  Citations

• Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy

  Elsa G Shapiro;W. Krivit;L. Lockman;I. Jambaqué

  404
  Citations

• Bone marrow-derived mesenchymal stem cells remain host-derived despite successful hematopoietic engraftment after allogeneic transplantation in patients with lysosomal and peroxisomal storage diseases

  Omer N. Koç;Charles Peters;Patrick Aubourg;Shrini Raghavan

  352
  Citations

• The gene responsible for adrenoleukodystrophy encodes a peroxisomal membrane protein

  Jean Mosser;Yves Lutz;Marie Elisabeth Stoeckel;Claude Olivier Sarde

  297
  Citations

• X-linked adrenoleukodystrophy: clinical, metabolic, genetic and pathophysiological aspects.

  Stephan Kemp;Johannes Berger;Patrick Aubourg

  290
  Citations

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

  Aubourg P;Adamsbaum C;Lavallard-Rousseau Mc;Rocchiccioli F

  289
  Citations

• Adeno-associated virus gene therapy with cholesterol 24-hydroxylase reduces the amyloid pathology before or after the onset of amyloid plaques in mouse models of Alzheimer's disease.

  Eloise Hudry;Debby Van Dam;Wim Kulik;Peter P De Deyn

  266
  Citations

• Case Definition and Classification of Leukodystrophies and Leukoencephalopathies

  Adeline Vanderver;Morgan Prust;Davide Tonduti;Fanny Mochel

  255
  Citations

• X-linked adrenoleukodystrophy in women: a cross-sectional cohort study

  Marc Engelen;Mathieu Barbier;Inge M. E. Dijkstra;Remmelt Schür

  240
  Citations

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

  Morgane Perdomini;Brahim Belbellaa;Laurent Monassier;Laurence Reutenauer

  237
  Citations

• Human CD34+ cells differentiate into microglia and express recombinant therapeutic protein.

  Muriel Asheuer;Françoise Pflumio;Sonia Benhamida;Anne Dubart-Kupperschmitt

  197
  Citations

• Homo- and heterodimerization of peroxisomal ATP-binding cassette half-transporters.

  Lang Xia Liu;Katy Janvier;Véronique Berteaux-Lecellier;Nathalie Cartier

  183
  Citations