Génétique et Biothérapies des maladies dégénératives du système nerveux et endocriniennes

Institution

Inserm
Mircen CEA

Laboratory

Neurosciences Bicêtre Paris Sud
Phone: +33 (0)1 49 59 53 50
U 986

Mots clefs

Alzheimer
Maladies neurodégénératives
therapie génique
leucodystrophies
cholesterol

publications

Cartier N, Hacein-Bey-Abina S, Bartholomä C, Veres G, Schmidt M, Kutschera I, Vidaud M, Dal-Cortivo L, Caccavelli L, Malhaoui N,Kiermer V, Mittelstaedt D, Bellesme C, Audat F, Blanche S, Audit M, l?Homme B, Bougnères P, Fischer A, Von Kalle C, Cavazana M,Aubourg P Hematopoietic Stem Cell Gene Therapy With Lentiviral Vector in Xadrenoleukodystrophy.Science, 2009 : 26(5954):818-23.

Marie-Anne Colle*, Françoise Piguet*, Lise Bertrand, Sylvie Raoul, Ivan Bieche, Laurence Dubreuil, Didi Sloothaak, Philippe Moullier,Patrick Aubourg, Yan Cherel, Caroline Sevin*, Nathalie Cartier*
Intracerebral delivery of AAV2-5 vector encoding human ARSA in non-human primates : transgene expression and bioactivity HumMol Genet. 19(1):147-58. 2010

Fourcade S, Ruiz M, Guilera C, Hahnen E, Brichta L, Naudi A, Portero-Otín M, Dacremont G, Cartier N, Wanders R, Kemp S, Mandel JL, Wirth B, Pamplona R, Aubourg P, Pujol A.Valproic acid induces antioxidant effects in X-linked Adrenoleukodystrophy.Hum Mol Genet. 2010 15;19(10):2005-14.

MARIE-ANNE COLLE*, FRANÇOISE PIGUET*, LISE BERTRAND, SYLVIE RAOUL, IVAN BIECHE, LAURENCE DUBREUIL, DIDI SLOOTHAAK, PHILIPPE MOULLIER, PATRICK AUBOURG, YAN CHEREL, CAROLINE SEVIN*, NATHALIE CARTIER* Intracerebral delivery of AAV2-5 vector encoding human ARSA in non-human primates : transgene expression and bioactivity HumMol Genet. 2010 Jan 1;19(1):147-58.

CAVAZZANA-CALVO M, PAYEN E, NEGRE O, WANG G, HEHIR K, FUSIL F, DOWN J, DENARO M, BRADY T, WESTERMAN K,CAVALLESCO R, GILLET-LEGRAND B, CACCAVELLI L, SGARRA R, MAOUCHE-CHRÉTIEN L, BERNAUDIN F, GIROT R,DORAZIO R, MULDER GJ, POLACK A, BANK A, SOULIER J, LARGHERO J, KABBARA N, DALLE B, GOURMEL B, SOCIE G,CHRÉTIEN S, CARTIER N, AUBOURG P, FISCHER A, CORNETTA K, GALACTEROS F, BEUZARD Y, GLUCKMAN E, BUSHMAN F, HACEIN-BEY-ABINA S, LEBOULCH P Transfusion independence and HMGA2 activation after gene therapy of human ?-thalassaemia.Nature. 2010 : 467 : 318-22

Fields of research

Neurological and psychiatric diseases

Research Theme

The NBPS (Neurosciences Bicêtre Paris Sud) brings together a clinical department of Paediatric Neurology (Pr. P. Aubourg), a clinical Department of adult Neurology and a clinical Department of Psychiatry associated with a Department of Interventional Neuroradiology, and a Department of Neurosurgery, supported by two platforms for research and clinical studies:

1. The Platform (P. Aubourg) for the clinical selection and clinical investigation of paediatric and adult CNS diseases that can be candidate for gene therapy approach.

 2. The Platform (N. Cartier as scientific responsible of CNS gene therapy) at U986 of INSERM to assess in animal and cell models preclinical gene therapy for these specific diseases.

The platform at U986 of INSERM is located at CEA-Fontenay-aux Roses and is part of the MIRCen platform directed by Philippe Hantraye. It includes: animal housing (rodents) operating under either BCL1 or BCL2), non-human primates housing operating under either BCL1, BCL2 or BCL3, neurosurgery for non-human primates, behavioural laboratory for rodents (motor & cognitive), neuropathological and biochemistry facilities, a laboratory dedicated to the biodistribution of viral vector into the brain and peripheral organs (3 high-throughput real-time PCR, one pyrosequencer). It benefitiates from the other infrastructures implemented at MIRCen: brain MRI using 7 Tesla (and soon 13 Tesla for rodents); PET, 3D neuropathological reconstruction, viral vector production.

Les pathologies pour lesquelles l'équipe a des projets de thérapie génique en cours (certains en collaboration avec d'autres groupes):

  • Forme cérébrale d'adrénoleucodystrophie
  • Forme médullaire d'adrénoleucodystrophie (adrénomyéloneuropathie)
  • Leucodystrophie métachromatiqueMaladie de Pelizaeus-Merzbacher (en collaboration avec Odile Boespflug-Tanguy, Université Paris VII)
  • Forme héréditaires de maladie d'Alzheimer
  • Tauopathies
  • Maladie de Huntington (en collaboration avec Jocelyne Caboche, UPMC)
  • Maladie de Friedreich (en collaboration avec Hélène Puccio, IGBMC, Strasbourg)



Membres de l'équipe

FRADIN Delphine