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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.
Journal articles
hal-01779044v1
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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.
Journal articles
cea-00905288v1
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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.
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.
Journal articles
hal-01299208v1
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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.
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.
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.
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.
Journal articles
hal-03228637v1
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