Aubourg, Patrick

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

Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., et al. (2009) · Science

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 1,508

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

Cavazzana-Calvo, M., Payen, E., Negre, O., Wang, G., Hehir, K., Fusil, F., Down, J., Denaro, M., Brady, T., Westerman, K., et al. (2010) · Nature

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 1,276

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

Mosser, J., Douar, A.-M., Sarde, C.-O., Kioschis, P., Feil, R., Moser, H., Poustka, A.-M., Mandel, J.-L., & Aubourg, P. (1993) · Nature

Metabolism and Genetic DisordersHereditary Neurological Disorders

Cited by 1,202

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

Muffat, J., Li, Y., Yuan, B., Mitalipova, M., Omer, A., Corcoran, S., Bakiasi, G., Tsai, L.-H., Aubourg, P., Ransohoff, R. M., et al. (2016) · Nature Medicine

Pluripotent Stem Cells ResearchNeuroinflammation and Neurodegeneration Mechanisms

Cited by 651

Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy

Eichler, F., Duncan, C., Musolino, P. L., Orchard, P. J., De Oliveira, S., Thrasher, A. J., Armant, M., Dansereau, C., Lund, T. C., Miller, W. P., et al. (2017) · New England Journal of Medicine

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 604

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

Engelen, M., Kemp, S., de Visser, M., van Geel, B. M., Wanders, R. J., Aubourg, P., & Poll-The, B. (2012) · Orphanet Journal of Rare Diseases

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 562

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

Peters, C., Charnas, L. R., Tan, Y., Ziegler, R. S., Shapiro, E. G., DeFor, T., Grewal, S. S., Orchard, P. J., Abel, S. L., Goldman, A. I., et al. (2004) · Blood

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 374

Reversal of Early Neurologic and Neuroradiologic Manifestations of X-Linked Adrenoleukodystrophy by Bone Marrow Transplantation

Aubourg, P., Blanche, S., Jambaqué, I., Rocchiccioli, F., Kalifa, G., Naud-Saudreau, C., Rolland, M.-O., Debré, M., Chaussain, J.-L., Griscelli, C., et al. (1990) · New England Journal of Medicine

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 366

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

Shapiro, E., Krivit, W., Lockman, L., Jambaqué, I., Peters, C., Cowan, M., Harris, R., Blanche, S., Bordigoni, P., Loes, D., et al. (2000) · The Lancet

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 329

X-linked adrenoleukodystrophy: Clinical, metabolic, genetic and pathophysiological aspects

Kemp, S., Berger, J., & Aubourg, P. (2012) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 283

The gene responsible for adrenoleukodystrophy encodes a peroxisomal membrane protein

Mosser, J., Lutz, Y., Stoeckel, M. E., Sarde, C. O., Kretz, C., Douar, A. M., Lopez, J., Aubourg, P., & Mandel, J. L. (1994) · Human Molecular Genetics

Metabolism and Genetic DisordersHereditary Neurological Disorders

Cited by 281

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

Aubourg, P., Adamsbaum, C., Lavallard-Rousseau, M.-C., Rocchiccioli, F., Cartier, N., Jambaque, I., Jakobezak, C., Lemaitre, A., Boureau, F., Wolf, C., et al. (1993) · New England Journal of Medicine

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 242

CYP46A1 inhibition, brain cholesterol accumulation and neurodegeneration pave the way for Alzheimer’s disease

Djelti, F., Braudeau, J., Hudry, E., Dhenain, M., Varin, J., Bièche, I., Marquer, C., Chali, F., Ayciriex, S., Auzeil, N., et al. (2015) · Brain

Alzheimer's disease research and treatmentsCholesterol and Lipid Metabolism

Cited by 223

Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy

Cartier, N., & Aubourg, P. (2010) · Brain Pathology

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 151

Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation

Raymond, G. V., Aubourg, P., Paker, A., Escolar, M., Fischer, A., Blanche, S., Baruchel, A., Dalle, J.-H., Michel, G., Prasad, V., et al. (2019) · Biology of Blood and Marrow Transplantation

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 142

Homo- and Heterodimerization of Peroxisomal ATP-binding Cassette Half-transporters

Liu, L. X., Janvier, K., Berteaux-Lecellier, V., Cartier, N., Benarous, R., & Aubourg, P. (1999) · Journal of Biological Chemistry

Metabolism and Genetic DisordersDrug Transport and Resistance Mechanisms

Cited by 141

General aspects and neuropathology of X-linked adrenoleukodystrophy

Ferrer, I., Aubourg, P., & Pujol, A. (2010) · Brain Pathology

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 139

Lentiviral Hematopoietic Cell Gene Therapy for X-Linked Adrenoleukodystrophy

Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Bougnères, P., Schmidt, M., Von Kalle, C., Fischer, A., Cavazzana-Calvo, M., & Aubourg, P. (2012) · Methods in Enzymology

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 123

The neurobiology of X-linked adrenoleukodystrophy, a demyelinating peroxisomal disorder

Dubois-Dalcq, M., Feigenbaum, V., & Aubourg, P. (1999) · Trends in Neurosciences

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 121

Expression of the adrenoleukodystrophy protein in the human and mouse central nervous system

Fouquet, F., Zhou, J. M., Ralston, E., Murray, K., Troalen, F., Magal, E., Robain, O., Dubois-Dalcq, M., & Aubourg, P. (1997) · Neurobiology of Disease

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 115

Oxidative stress underlying axonal degeneration in adrenoleukodystrophy: A paradigm for multifactorial neurodegenerative diseases?

Galea, E., Launay, N., Portero-Otin, M., Ruiz, M., Pamplona, R., Aubourg, P., Ferrer, I., & Pujol, A. (2012) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease

Metabolism and Genetic DisordersNeurological diseases and metabolism

Cited by 108

X-linked adrenoleukodystrophy is a frequent cause of idiopathic Addison's disease in young adult male patients.

Laureti, S., Casucci, G., Santeusanio, F., Angeletti, G., Aubourg, P., & Brunetti, P. (1996) · The Journal of Clinical Endocrinology & Metabolism

Metabolism and Genetic DisordersAdrenal Hormones and Disorders

Cited by 101

Gene therapy for metachromatic leukodystrophy

Rosenberg, J. B., Kaminsky, S. M., Aubourg, P., Crystal, R. G., & Sondhi, D. (2016) · Journal of Neuroscience Research

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 91

Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice

Piguet, F., Sondhi, D., Piraud, M., Fouquet, F., Hackett, N. R., Ahouansou, O., Vanier, M.-T., Bieche, I., Aubourg, P., Crystal, R. G., et al. (2012) · Human Gene Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 91