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Items: 9


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.


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.


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.


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.


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.


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.


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