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Items: 41


Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice.

Niemir N, Rouvière L, Besse A, Vanier MT, Dmytrus J, Marais T, Astord S, Puech JP, Panasyuk G, Cooper JD, Barkats M, Caillaud C.

Hum Mol Genet. 2018 Mar 15;27(6):954-968. doi: 10.1093/hmg/ddy012.


Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F.

Sci Transl Med. 2017 Nov 29;9(418). pii: eaam6375. doi: 10.1126/scitranslmed.aam6375.


A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.

Biferi MG, Cohen-Tannoudji M, Cappelletto A, Giroux B, Roda M, Astord S, Marais T, Bos C, Voit T, Ferry A, Barkats M.

Mol Ther. 2017 Sep 6;25(9):2038-2052. doi: 10.1016/j.ymthe.2017.05.017. Epub 2017 Jun 26.


β-Amyloid Precursor Protein Intracellular Domain Controls Mitochondrial Function by Modulating Phosphatase and Tensin Homolog-Induced Kinase 1 Transcription in Cells and in Alzheimer Mice Models.

Goiran T, Duplan E, Chami M, Bourgeois A, El Manaa W, Rouland L, Dunys J, Lauritzen I, You H, Stambolic V, Biféri MG, Barkats M, Pimplikar SW, Sergeant N, Colin M, Morais VA, Pardossi-Piquard R, Checler F, Alves da Costa C.

Biol Psychiatry. 2018 Mar 1;83(5):416-427. doi: 10.1016/j.biopsych.2017.04.011. Epub 2017 May 3.


SECIS-binding protein 2 interacts with the SMN complex and the methylosome for selenoprotein mRNP assembly and translation.

Gribling-Burrer AS, Leichter M, Wurth L, Huttin A, Schlotter F, Troffer-Charlier N, Cura V, Barkats M, Cavarelli J, Massenet S, Allmang C.

Nucleic Acids Res. 2017 May 19;45(9):5399-5413. doi: 10.1093/nar/gkx031.


A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome.

Matagne V, Ehinger Y, Saidi L, Borges-Correia A, Barkats M, Bartoli M, Villard L, Roux JC.

Neurobiol Dis. 2017 Mar;99:1-11. doi: 10.1016/j.nbd.2016.12.009. Epub 2016 Dec 11.


Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.

Armbruster N, Lattanzi A, Jeavons M, Van Wittenberghe L, Gjata B, Marais T, Martin S, Vignaud A, Voit T, Mavilio F, Barkats M, Buj-Bello A.

Mol Ther Methods Clin Dev. 2016 Sep 14;3:16060. doi: 10.1038/mtm.2016.60. eCollection 2016.


Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins.

Alves S, Marais T, Biferi MG, Furling D, Marinello M, El Hachimi K, Cartier N, Ruberg M, Stevanin G, Brice A, Barkats M, Sittler A.

Mol Neurodegener. 2016 Jul 28;11(1):58. doi: 10.1186/s13024-016-0123-2.


Intraneuronal aggregation of the β-CTF fragment of APP (C99) induces Aβ-independent lysosomal-autophagic pathology.

Lauritzen I, Pardossi-Piquard R, Bourgeois A, Pagnotta S, Biferi MG, Barkats M, Lacor P, Klein W, Bauer C, Checler F.

Acta Neuropathol. 2016 Aug;132(2):257-276. doi: 10.1007/s00401-016-1577-6. Epub 2016 Apr 30.


Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases.

Dragin N, Bismuth J, Cizeron-Clairac G, Biferi MG, Berthault C, Serraf A, Nottin R, Klatzmann D, Cumano A, Barkats M, Le Panse R, Berrih-Aknin S.

J Clin Invest. 2016 Apr 1;126(4):1525-37. doi: 10.1172/JCI81894. Epub 2016 Mar 21.


Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice.

Tanguy Y, Biferi MG, Besse A, Astord S, Cohen-Tannoudji M, Marais T, Barkats M.

Front Mol Neurosci. 2015 Jul 28;8:36. doi: 10.3389/fnmol.2015.00036. eCollection 2015.


Moving towards treatments for spinal muscular atrophy: hopes and limits.

Wirth B, Barkats M, Martinat C, Sendtner M, Gillingwater TH.

Expert Opin Emerg Drugs. 2015 Sep;20(3):353-6. doi: 10.1517/14728214.2015.1041375. Epub 2015 Jul 3. Review.


The autophagy/lysosome pathway is impaired in SCA7 patients and SCA7 knock-in mice.

Alves S, Cormier-Dequaire F, Marinello M, Marais T, Muriel MP, Beaumatin F, Charbonnier-Beaupel F, Tahiri K, Seilhean D, El Hachimi K, Ruberg M, Stevanin G, Barkats M, den Dunnen W, Priault M, Brice A, Durr A, Corvol JC, Sittler A.

Acta Neuropathol. 2014 Nov;128(5):705-22. doi: 10.1007/s00401-014-1289-8. Epub 2014 May 24.


scAAV9 intracisternal delivery results in efficient gene transfer to the central nervous system of a feline model of motor neuron disease.

Bucher T, Colle MA, Wakeling E, Dubreil L, Fyfe J, Briot-Nivard D, Maquigneau M, Raoul S, Cherel Y, Astord S, Duque S, Marais T, Voit T, Moullier P, Barkats M, Joussemet B.

Hum Gene Ther. 2013 Jul;24(7):670-82. doi: 10.1089/hum.2012.218.


A single intravenous AAV9 injection mediates bilateral gene transfer to the adult mouse retina.

Bemelmans AP, Duqué S, Rivière C, Astord S, Desrosiers M, Marais T, Sahel JA, Voit T, Barkats M.

PLoS One. 2013 Apr 15;8(4):e61618. doi: 10.1371/journal.pone.0061618. Print 2013.


Interferon β induces clearance of mutant ataxin 7 and improves locomotion in SCA7 knock-in mice.

Chort A, Alves S, Marinello M, Dufresnois B, Dornbierer JG, Tesson C, Latouche M, Baker DP, Barkats M, El Hachimi KH, Ruberg M, Janer A, Stevanin G, Brice A, Sittler A.

Brain. 2013 Jun;136(Pt 6):1732-45. doi: 10.1093/brain/awt061. Epub 2013 Mar 21.


Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice.

Benkhelifa-Ziyyat S, Besse A, Roda M, Duque S, Astord S, Carcenac R, Marais T, Barkats M.

Mol Ther. 2013 Feb;21(2):282-90. doi: 10.1038/mt.2012.261. Epub 2013 Jan 8.


Implication of the SMN complex in the biogenesis and steady state level of the signal recognition particle.

Piazzon N, Schlotter F, Lefebvre S, Dodré M, Méreau A, Soret J, Besse A, Barkats M, Bordonné R, Branlant C, Massenet S.

Nucleic Acids Res. 2013 Jan;41(2):1255-72. doi: 10.1093/nar/gks1224. Epub 2012 Dec 5.


A rare recessive distal hereditary motor neuropathy with HSJ1 chaperone mutation.

Blumen SC, Astord S, Robin V, Vignaud L, Toumi N, Cieslik A, Achiron A, Carasso RL, Gurevich M, Braverman I, Blumen N, Munich A, Barkats M, Viollet L.

Ann Neurol. 2012 Apr;71(4):509-19. doi: 10.1002/ana.22684.


Failure of lower motor neuron radial outgrowth precedes retrograde degeneration in a feline model of spinal muscular atrophy.

Wakeling EN, Joussemet B, Costiou P, Fanuel D, Moullier P, Barkats M, Fyfe JC.

J Comp Neurol. 2012 Jun 1;520(8):1737-50. doi: 10.1002/cne.23010.


Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice.

Dominguez E, Marais T, Chatauret N, Benkhelifa-Ziyyat S, Duque S, Ravassard P, Carcenac R, Astord S, Pereira de Moura A, Voit T, Barkats M.

Hum Mol Genet. 2011 Feb 15;20(4):681-93. doi: 10.1093/hmg/ddq514. Epub 2010 Nov 30.


Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.

Duque S, Joussemet B, Riviere C, Marais T, Dubreil L, Douar AM, Fyfe J, Moullier P, Colle MA, Barkats M.

Mol Ther. 2009 Jul;17(7):1187-96. doi: 10.1038/mt.2009.71. Epub 2009 Apr 14.


Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution.

Ciron C, Cressant A, Roux F, Raoul S, Cherel Y, Hantraye P, Déglon N, Schwartz B, Barkats M, Heard JM, Tardieu M, Moullier P, Colle MA.

Hum Gene Ther. 2009 Apr;20(4):350-60. doi: 10.1089/hum.2008.155.


The nuclear factor kappaB-activator gene PLEKHG5 is mutated in a form of autosomal recessive lower motor neuron disease with childhood onset.

Maystadt I, Rezsöhazy R, Barkats M, Duque S, Vannuffel P, Remacle S, Lambert B, Najimi M, Sokal E, Munnich A, Viollet L, Verellen-Dumoulin C.

Am J Hum Genet. 2007 Jul;81(1):67-76. Epub 2007 May 16.


Lentiviral vectors with a defective integrase allow efficient and sustained transgene expression in vitro and in vivo.

Philippe S, Sarkis C, Barkats M, Mammeri H, Ladroue C, Petit C, Mallet J, Serguera C.

Proc Natl Acad Sci U S A. 2006 Nov 21;103(47):17684-9. Epub 2006 Nov 9.


1-methyl-4-phenylpyridinium neurotoxicity is attenuated by adenoviral gene transfer of human Cu/Zn superoxide dismutase.

Barkats M, Horellou P, Colin P, Millecamps S, Faucon-Biguet N, Mallet J.

J Neurosci Res. 2006 Feb 1;83(2):233-42.


Synaptic sprouting increases the uptake capacities of motoneurons in amyotrophic lateral sclerosis mice.

Millecamps S, Nicolle D, Ceballos-Picot I, Mallet J, Barkats M.

Proc Natl Acad Sci U S A. 2001 Jun 19;98(13):7582-7. Epub 2001 Jun 12.


Overexpression of glutathione peroxidase increases the resistance of neuronal cells to Abeta-mediated neurotoxicity.

Barkats M, Millecamps S, Abrioux P, Geoffroy MC, Mallet J.

J Neurochem. 2000 Oct;75(4):1438-46.


Neuron-restrictive silencer elements mediate neuron specificity of adenoviral gene expression.

Millecamps S, Kiefer H, Navarro V, Geoffroy MC, Robert JJ, Finiels F, Mallet J, Barkats M.

Nat Biotechnol. 1999 Sep;17(9):865-9.


Adenovirus in the brain: recent advances of gene therapy for neurodegenerative diseases.

Barkats M, Bilang-Bleuel A, Buc-Caron MH, Castel-Barthe MN, Corti O, Finiels F, Horellou P, Revah F, Sabate O, Mallet J.

Prog Neurobiol. 1998 Jul;55(4):333-41. Review.


Effects of Ginkgo biloba extract (EGb 761) on learning and possible actions on aging.

Cohen-Salmon C, Venault P, Martin B, Raffalli-Sébille MJ, Barkats M, Clostre F, Pardon MC, Christen Y, Chapouthier G.

J Physiol Paris. 1997 Dec;91(6):291-300.


Intrastriatal grafts of embryonic mesencephalic rat neurons genetically modified using an adenovirus encoding human Cu/Zn superoxide dismutase.

Barkats M, Nakao N, Grasbon-Frodl EM, Bilang-Bleuel A, Revah F, Mallet J, Brundin P.

Neuroscience. 1997 Jun;78(3):703-13.


Age-related morphological changes in the hippocampus in two mouse strains.

Barkats M, Bertholet JY, Cohen-Salmon C.

Mech Ageing Dev. 1996 Jun 25;87(3):155-64.


An adenovirus encoding CuZnSOD protects cultured striatal neurones against glutamate toxicity.

Barkats M, Bemelmans AP, Geoffroy MC, Robert JJ, Loquet I, Horellou P, Revah F, Mallet J.

Neuroreport. 1996 Jan 31;7(2):497-501.


Specific and efficient gene transfer strategy offers new potentialities for the treatment of motor neurone diseases.

Finiels F, Gimenez y Ribotta M, Barkats M, Samolyk ML, Robert JJ, Privat A, Revah F, Mallet J.

Neuroreport. 1995 Dec 29;7(1):373-8.


Specific and efficient gene transfer strategy offers new potentialities for the treatment of motor neurone diseases.

Finiels F, Gimenez y Ribotta M, Barkats M, Samolyk ML, Robert JJ, Privat A, Revah F, Mallet J.

Neuroreport. 1995 Dec 15;6(18):2473-8.


Adenovirus for neurodegenerative diseases: in vivo strategies and ex vivo gene therapy using human neural progenitors.

Sabaté O, Barkats M, Buc-Caron MH, Castel-Barthe MN, Finiels F, Horellou P, Revah F, Mallet J.

Clin Neurosci. 1995-1996;3(5):317-21. Review.


Hippocampal mossy fiber changes in mice transgenic for the human copper-zinc superoxide dismutase gene.

Barkats M, Bertholet JY, Venault P, Ceballos-Picot I, Nicole A, Phillips J, Moutier R, Roubertoux P, Sinet PM, Cohen-Salmon C.

Neurosci Lett. 1993 Sep 17;160(1):24-8.


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