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authFullName_s : Patrick Aubourg

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

Sophie Ayciriex , Fathia Djelti , Jérome Braudeau , Eloise Hudry , Marc Dhenain , et al.
Brain - A Journal of Neurology , 2015, 138 (8), pp.2383 - 2398. ⟨10.1093/brain/awv166⟩
Journal articles hal-01779044v1

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

Marina Cavazzana-Calvo , Emmanuel Payen , Olivier Negre , Gary Wang , Kathleen Hehir , et al.
Nature, 2010, 467 (7313), pp.318-322. ⟨10.1038/nature09328⟩
Journal articles cea-00905288v1

Intracerebral gene therapy in children with metachromatic leukodystrophy: Results of a phase I/II trial

Caroline Sevin , Thomas Roujeau , Nathalie Cartier , Thomas Baugnon , Catherine Adamsbaum , et al.
14. Annual WORLD Symposium (WORLD Symposium 2018), Feb 2018, San Diego, Californie, United States. 173 p., ⟨10.1016/j.ymgme.2017.12.352⟩
Conference papers hal-02737676v1
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CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington’s disease

Lydie Boussicault , Sandro Alves , Antonin Lamazière , Anabelle Planques , Nicolas Heck , et al.
Brain - A Journal of Neurology , 2016, 139 (3), pp.953-970. ⟨10.1093/brain/awv384⟩
Journal articles hal-01299208v1

Neuronal Cholesterol Accumulation Induced by Cyp46a1 Down-Regulation in Mouse Hippocampus Disrupts Brain Lipid Homeostasis

Sophie Ayciriex , Fathia Djelti , Sandro Alves , Anne Regazzetti , Mathieu Gaudin , et al.
Frontiers in Molecular Neuroscience, 2017, 10, ⟨10.3389/fnmol.2017.00211⟩
Journal articles hal-03220550v1
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Hematopoietic stem cell Gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

Nathalie Cartier , Salima Hacein-Bey-Abina , Cynthia C. Bartholomae , Gabor Veres , Manfred Schmidt , et al.
Science, 2009, 326 (5954), pp.818-823. ⟨10.1126/science.1171242⟩
Journal articles cea-00817492v1
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Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?

Karim Bey , Johan Deniaud , Laurence Dubreil , Béatrice Joussemet , Joseph Cristini , et al.
Molecular Therapy - Methods and Clinical Development, 2020, 17, pp.771-784. ⟨10.1016/j.omtm.2020.04.001⟩
Journal articles hal-02561949v1
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AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A

Benoit Gautier , Helene Hajjar , Sylvia Soares , Jade Berthelot , Marie Deck , et al.
Nature Communications, 2021, 12 (1), pp.2356. ⟨10.1038/s41467-021-22593-3⟩
Journal articles hal-03228637v1