Search Results - "Lostal, W."

High expression and therapeutic efficacy of systemic delivery of a dual AAV strategy in a murine model for dysferlin deficiency by Pryadkina, M, Roudaut, C, Charton, K, Lostal, W, Hirsch, M, Bourg, N, Richard, I

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T.O.2 Efficient recovery of dysferlin deficiency by dual adeno associated vector mediated gene transfer by Lostal, W, Bartoli, M, Bourg, N, Roudaut, C, Bentaib, A, Guerchet, N, Fougerousse, F, McNeil, P, Richard, I

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G.O.5 Partial functionality of a Mini-dysferlin molecule identified in a patient affected with moderately severe primary dysferlinopathy by Krahn, M, Wein, N, Lostal, W, Bourg-Alibert, N, Nguyen, K, Courrier, S, Vial, C, Labelle, V, Petris, D. De, Borges, A, Mattei, M.G, Roudaut, C, Miyake, K, McNeil, P, Cau, P, Leturcq, F, Bartoli, M, Lévy, N, Richard, I

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G.P.4.10 Functional evaluation of a putative mini-dysferlin identified in a patient with moderate Miyoshi myopathy phenotype by Krahn, M, Wein, N, Nguyen, K, Vial, C, Courrier, S, Lostal, W, Bartoli, M, Labelle, V, Leturcq, F, Cau, P, Richard, I, Levy, N

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OTHER NMDs by Biquand, A., Spinozzi, S., Tonino, P., Strom, J., Cosette, J., Elbeck, Z., Knöll, R., Granzier, H., Lostal, W., Richard, I.

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OTHER NMDs: EP.360 Is7 domain of titin is essential for cardiac function in mice by Biquand, A., Spinozzi, S., Tonino, P., Strom, J., Cosette, J., Elbeck, Z., Knöll, R., Granzier, H., Lostal, W., Richard, I.

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Establishment of a panel for the evaluation of the dystrophic process by quantitative RT-PCR by Vaubourg, C., Bellec, J., Gicquel, E., Lostal, W., Richard, I.

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P.236 - Establishment of a panel for the evaluation of the dystrophic process by quantitative RT-PCR by Vaubourg, C., Bellec, J., Gicquel, E., Lostal, W., Richard, I.

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Titin M-line insertion sequence 7 is required for proper cardiac function in mice by Biquand, Ariane, Spinozzi, Simone, Tonino, Paola, Cosette, Jérémie, Strom, Joshua, Elbeck, Zaher, Knöll, Ralph, Granzier, Henk, Lostal, William, Richard, Isabelle

“…Titin is a giant sarcomeric protein that is involved in a large number of functions, with a primary role in skeletal and cardiac sarcomere organization and…”
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P.156 - Preclinical studies for calpain 3 gene transfer: primate pilot study and dose effect evaluation by Lostal, W., Roudaut, C., Guilloux, V., Moullec, S., Larcher, T., Deschamps, J., Richard, I.

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Development of a CRISPR-based gene therapy approach targeting the large exon 45-exon 55 mutation hotspot in DMD gene by Bellec-Dyèvre, J., Lostal, W., Bernard, E., Cosette, J., Richard, I.

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P.298 - Development of a CRISPR-based gene therapy approach targeting the large exon 45-exon 55 mutation hotspot in DMD gene by Bellec-Dyèvre, J., Lostal, W., Bernard, E., Cosette, J., Richard, I.

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Preclinical studies for calpain 3 gene transfer: primate pilot study and dose effect evaluation by Lostal, W., Roudaut, C., Guilloux, V., Moullec, S., Larcher, T., Deschamps, J., Richard, I.

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G.P.157 - High expression and therapeutic efficacy of systemic delivery of a dual AAV strategy in a murine model for dysferlin deficiency by Pryadkina, M., Roudaut, C., Charton, K., Lostal, W., Hirsch, M., Bourg, N., Richard, I.

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Is7 domain of titin is essential for cardiac function in mice by Biquand, A, Spinozzi, S, Tonino, P, Strom, J, Cosette, J, Elbeck, Z, Knoll, R, Granzier, H, Lostal, W, Richard, I

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