Search Results - "Biferi, M."

Modeling and pathophysiological analysis of the ubiquilin-2-linked amyotrophic lateral sclerosis (ALS) with AAV vectors by Bos, C, Biferi, M, Cohen-Tannoudji, M, Roda, M, Barkats, M

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HEREDITARY NEUROPATHIES & ALS: P.110 Development and characterization of in vitro models to test the efficiency of gene therapy approaches in SOD1-linked Amyotrophic lateral sclerosis by Delamare, M., Elouej, S., Bigot, A., Cappella, M., Biferi, M.

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HEREDITARY NEUROPATHIES & ALS by Delamare, M., Elouej, S., Bigot, A., Cappella, M., Biferi, M.

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P.91Optimization of AAV-mediated gene therapy for SOD1-linked ALS by Biferi, M., Cohen-Tannoudji, M., Marais, T., Giroux, B., Astord, S.

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HEREDITARY NEUROPATHIES & ALS by Elouej, S., Delamare, M., Cappella, M., Cohen-Tannoudji, M., Astord, S., Marais, T., Giroux, B., Pezet, S., Biferi, M.

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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 by Elouej, S., Delamare, M., Cappella, M., Cohen-Tannoudji, M., Astord, S., Marais, T., Giroux, B., Pezet, S., Biferi, M.

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DISTAL MYOPATHIES by Elouej, S., Nelson, I., Cohen, E., Yaou, R. Ben, Isapof, A., Dubourg, O., Romero, N., Bonne, G., Biferi, M., Stojkovic, T.

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DISTAL MYOPATHIES: EP.75 Functional validation of a novel variant of the SPTAN1 gene identified in a family with distal motor myopathy with nerve involvement by Elouej, S., Nelson, I., Cohen, E., Yaou, R. Ben, Isapof, A., Dubourg, O., Romero, N., Bonne, G., Biferi, M., Stojkovic, T.

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AAV9-mediated SMN expression restricted to the CNS does not rescue SMA mice by Besse, A., Roda, M., Astord, S., Marais, T., Biferi, M., Barkats, M.

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P.132 - AAV9-mediated SMN expression restricted to the CNS does not rescue SMA mice by Besse, A., Roda, M., Astord, S., Marais, T., Biferi, M., Barkats, M.

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G.P.133 - Modeling and pathophysiological analysis of the ubiquilin-2-linked amyotrophic lateral sclerosis (ALS) with AAV vectors by Bos, C., Biferi, M., Cohen-Tannoudji, M., Roda, M., Barkats, M.

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TH.O.16 - A new AAV10-mediated gene therapy for SOD1-linked ALS by Biferi, M., Cohen-Tannoudji, M., Cappelletto, A., Giroux, B., Roda, M., Astord, S., Marais, T., Ferry, A., Voit, T., Barkats, M.

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A new AAV10-mediated gene therapy for SOD1 -linked ALS by Biferi, M., Cohen-Tannoudji, M., Cappelletto, A., Giroux, B., Roda, M., Astord, S., Marais, T., Ferry, A., Voit, T., Barkats, M.

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TH.O.17 - Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes by Colella, P., Puzzo, F., Biferi, M., Bali, D., Paulk, N., Vidal, P., Collaud, F., Simon-Sola, M., Charles, S., Hardet, R., Leborgne, C., Sellier, P., van Wittenberghe, L., Boisgerault, F., Barkats, M., Laforêt, P., Kay, M., Koeberl, D., Ronzitti, G., Mingozzi, F.

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Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes by Colella, P., Puzzo, F., Biferi, M., Bali, D., Paulk, N., Vidal, P., Collaud, F., Simon-Sola, M., Charles, S., Hardet, R., Leborgne, C., Sellier, P., van Wittenberghe, L., Boisgerault, F., Barkats, M., Laforêt, P., Kay, M., Koeberl, D., Ronzitti, G., Mingozzi, F.

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