Barkats M - Search Results

1

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. Niemir N, et al. Among authors: barkats m. Hum Mol Genet. 2018 Mar 15;27(6):954-968. doi: 10.1093/hmg/ddy012. Hum Mol Genet. 2018. PMID: 29325092

2

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. Dominguez E, et al. Among authors: barkats m. Hum Mol Genet. 2011 Feb 15;20(4):681-93. doi: 10.1093/hmg/ddq514. Epub 2010 Nov 30. Hum Mol Genet. 2011. PMID: 21118896

3

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. Benkhelifa-Ziyyat S, et al. Among authors: barkats m. Mol Ther. 2013 Feb;21(2):282-90. doi: 10.1038/mt.2012.261. Epub 2013 Jan 8. Mol Ther. 2013. PMID: 23295949 Free PMC article.

4

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. Tanguy Y, et al. Among authors: barkats m. Front Mol Neurosci. 2015 Jul 28;8:36. doi: 10.3389/fnmol.2015.00036. eCollection 2015. Front Mol Neurosci. 2015. PMID: 26283910 Free PMC article.

5

AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice.

Besse A, Astord S, Marais T, Roda M, Giroux B, Lejeune FX, Relaix F, Smeriglio P, Barkats M, Biferi MG. Besse A, et al. Among authors: barkats m. Mol Ther. 2020 Aug 5;28(8):1887-1901. doi: 10.1016/j.ymthe.2020.05.011. Epub 2020 May 15. Mol Ther. 2020. PMID: 32470325 Free PMC article.

6

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. Bucher T, et al. Among authors: barkats m. Hum Gene Ther. 2013 Jul;24(7):670-82. doi: 10.1089/hum.2012.218. Hum Gene Ther. 2013. PMID: 23799774

7

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. Biferi MG, et al. Among authors: barkats m. Mol Ther. 2017 Sep 6;25(9):2038-2052. doi: 10.1016/j.ymthe.2017.05.017. Epub 2017 Jun 26. Mol Ther. 2017. PMID: 28663100 Free PMC article.

8

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. Bemelmans AP, et al. Among authors: barkats m. PLoS One. 2013 Apr 15;8(4):e61618. doi: 10.1371/journal.pone.0061618. Print 2013. PLoS One. 2013. PMID: 23613884 Free PMC article.

9

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. Alves S, et al. Among authors: barkats m. Mol Neurodegener. 2016 Jul 28;11(1):58. doi: 10.1186/s13024-016-0123-2. Mol Neurodegener. 2016. PMID: 27465358 Free PMC article.

10

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. Armbruster N, et al. Among authors: barkats m. Mol Ther Methods Clin Dev. 2016 Sep 14;3:16060. doi: 10.1038/mtm.2016.60. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27652289 Free PMC article.

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