P3.10 Intramuscular AAV9 administration enables transgene delivery to motor neurons in the whole spinal cord: Therapeutic application in a SMA mouse model
Benkhelifa-Ziyyat, S, Besse, A, Duqué, S, Carcenac, R, Marais, T, Astord, S, Roda, M, Barkats, M
Published in Neuromuscular disorders : NMD (01.10.2011)
Published in Neuromuscular disorders : NMD (01.10.2011)
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O.18 Intravenous injection of SMN1-expressing self-complementary AAV9 rescues severe type I SMA mice
Dominguez, E, Chatauret, N, Marais, T, Duque, S, Carcenac, R, Astord, S, de Moura, A. Pereira, Voit, T, Barkats, M
Published in Neuromuscular disorders : NMD (01.10.2010)
Published in Neuromuscular disorders : NMD (01.10.2010)
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178P Spinal muscular atrophy type II skeletal muscle treated with Nusinersen and Risdiplam shows SMN restoration but mitochondrial deficiency
Grandi, F., Astord, S., Pezet, S., Gidaja, E., Mazzucchi, S., Chapart, M., Vasseur, S., Mamchaoui, K., Smeriglio, P.
Published in Neuromuscular disorders : NMD (01.10.2024)
Published in Neuromuscular disorders : NMD (01.10.2024)
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177P Effect of gene therapy on the epigenetic and transcriptional stability of SMA
Smeriglio, P., Grandi, F., Arnould, A., Pezet, S., Marais, T., Mazzucchi, S., Astord, S., Cohen-Tannoudji, M.
Published in Neuromuscular disorders : NMD (01.10.2024)
Published in Neuromuscular disorders : NMD (01.10.2024)
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Journal Article
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
Puzzo, Francesco, Colella, Pasqualina, Biferi, Maria G, Bali, Deeksha, Paulk, Nicole K, Vidal, Patrice, Collaud, Fanny, Simon-Sola, Marcelo, Charles, Severine, Hardet, Romain, Leborgne, Christian, Meliani, Amine, Cohen-Tannoudji, Mathilde, Astord, Stephanie, Gjata, Bernard, Sellier, Pauline, van Wittenberghe, Laetitia, Vignaud, Alban, Boisgerault, Florence, Barkats, Martine, Laforet, Pascal, Kay, Mark A, Koeberl, Dwight D, Ronzitti, Giuseppe, Mingozzi, Federico
Published in Science translational medicine (29.11.2017)
Published in Science translational medicine (29.11.2017)
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P.91Optimization of AAV-mediated gene therapy for SOD1-linked ALS
Biferi, M., Cohen-Tannoudji, M., Marais, T., Giroux, B., Astord, S.
Published in Neuromuscular disorders : NMD (01.10.2019)
Published in Neuromuscular disorders : NMD (01.10.2019)
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Journal Article
HEREDITARY NEUROPATHIES & ALS
Elouej, S., Delamare, M., Cappella, M., Cohen-Tannoudji, M., Astord, S., Marais, T., Giroux, B., Pezet, S., Biferi, M.
Published in Neuromuscular disorders : NMD (01.10.2020)
Published in Neuromuscular disorders : NMD (01.10.2020)
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HEREDITARY NEUROPATHIES & ALS: P.115 Analysis of off-target effects of antisense sequence inducing exon skipping in SOD1-linked amyotrophic lateral sclerosis
Elouej, S., Delamare, M., Cappella, M., Cohen-Tannoudji, M., Astord, S., Marais, T., Giroux, B., Pezet, S., Biferi, M.
Published in Neuromuscular disorders : NMD (01.10.2020)
Published in Neuromuscular disorders : NMD (01.10.2020)
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AAV9-mediated SMN expression restricted to the CNS does not rescue SMA mice
Besse, A., Roda, M., Astord, S., Marais, T., Biferi, M., Barkats, M.
Published in Neuromuscular disorders : NMD (01.10.2017)
Published in Neuromuscular disorders : NMD (01.10.2017)
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Journal Article
P.131 - Role of muscle satellite cells in Spinal muscular atrophy physiopathology
Mecca, J., Astord, S., Marais, T., Relaix, F., Didier, N., Barkats, M.
Published in Neuromuscular disorders : NMD (01.10.2017)
Published in Neuromuscular disorders : NMD (01.10.2017)
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Journal Article
Role of muscle satellite cells in Spinal muscular atrophy physiopathology
Mecca, J., Astord, S., Marais, T., Relaix, F., Didier, N., Barkats, M.
Published in Neuromuscular disorders : NMD (01.10.2017)
Published in Neuromuscular disorders : NMD (01.10.2017)
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Journal Article
P.132 - AAV9-mediated SMN expression restricted to the CNS does not rescue SMA mice
Besse, A., Roda, M., Astord, S., Marais, T., Biferi, M., Barkats, M.
Published in Neuromuscular disorders : NMD (01.10.2017)
Published in Neuromuscular disorders : NMD (01.10.2017)
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TH.O.16 - A new AAV10-mediated gene therapy for SOD1-linked ALS
Biferi, M., Cohen-Tannoudji, M., Cappelletto, A., Giroux, B., Roda, M., Astord, S., Marais, T., Ferry, A., Voit, T., Barkats, M.
Published in Neuromuscular disorders : NMD (01.10.2017)
Published in Neuromuscular disorders : NMD (01.10.2017)
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A new AAV10-mediated gene therapy for SOD1 -linked ALS
Biferi, M., Cohen-Tannoudji, M., Cappelletto, A., Giroux, B., Roda, M., Astord, S., Marais, T., Ferry, A., Voit, T., Barkats, M.
Published in Neuromuscular disorders : NMD (01.10.2017)
Published in Neuromuscular disorders : NMD (01.10.2017)
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