Cartier, Nathalie

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

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Hocquemiller, M., Giersch, L., Audrain, M., Parker, S., & Cartier, N. (2016) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 291

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

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

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

Retroviral-mediated gene transfer corrects very-long-chain fatty acid metabolism in adrenoleukodystrophy fibroblasts.

Cartier, N., Lopez, J., Moullier, P., Rocchiccioli, F., Rolland, M. O., Jorge, P., Mosser, J., Mandel, J. L., Bougnères, P. F., & Danos, O. (1995) · Proceedings of the National Academy of Sciences

Virus-based gene therapy researchMetabolism and Genetic Disorders

Cited by 105

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

Enzyme, cell and gene‐based therapies for metachromatic leukodystrophy

Sevin, C., Aubourg, P., & Cartier, N. (2007) · Journal of Inherited Metabolic Disease

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 88

The APP Intracellular Domain Is Required for Normal Synaptic Morphology, Synaptic Plasticity, and Hippocampus-Dependent Behavior

Klevanski, M., Herrmann, U., Weyer, S. W., Fol, R., Cartier, N., Wolfer, D. P., Caldwell, J. H., Korte, M., & Müller, U. C. (2015) · The Journal of Neuroscience

Alzheimer's disease research and treatmentsNeuroscience and Neuropharmacology Research

Cited by 83

Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice

Benhamida, S., Pflumio, F., Dubart-Kupperschmitt, A., Zhao-Emonet, J.-C., Cavazzana-Calvo, M., Rocchiccioli, F., Fichelson, S., Aubourg, P., Charneau, P., & Cartier, N. (2003) · Molecular Therapy

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 61

Abnormal messenger RNA expression and a missense mutation in patients with X-linked adrenoleukodystrophy

Cartier, N., Sarde, C.-O., Douar, A.-M., Mosser, J., Mandel, J.-L., & Aubourg, P. (1993) · Human Molecular Genetics

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 55

Gene Therapy Strategies for Alzheimer's Disease: An Overview

Alves, S., Fol, R., & Cartier, N. (2016) · Human Gene Therapy

Alzheimer's disease research and treatmentsVirus-based gene therapy research

Cited by 45

Modulation of Brain Cholesterol Metabolism through CYP46A1 Overexpression for Rett Syndrome

Audouard, E., Khefif, N., Gillet-Legrand, B., Nobilleau, F., Bouazizi, O., Stanga, S., Despres, G., Alves, S., Lamazière, A., Cartier, N., et al. (2024) · Pharmaceutics

Genetics and Neurodevelopmental DisordersVirus-based gene therapy research

Cited by 7