Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model

Audrey Bourdon; Virginie François; Liwen Zhang; Aude Lafoux; Bodvael Fraysse; Gilles Toumaniantz; Thibaut Larcher; Tiphaine Girard; Mireille Ledevin; Cyrielle Lebreton; Agnès Hivonnait; Anna Creismeas; Marine Allais; Basile Marie; Justine Guguin; Véronique Blouin; Séverine Remy; Ignacio Anegon; Corinne Huchet; Alberto Malerba; Betty Kao; Anita Le Heron; Philippe Moullier; George Dickson; Linda Popplewell; Oumeya Adjali; Federica Montanaro; Caroline Le Guiner

doi:10.1038/s41434-022-00317-6

Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model

Audrey Bourdon, Virginie François, Liwen Zhang, Aude Lafoux, Bodvael Fraysse, Gilles Toumaniantz, Thibaut Larcher, Tiphaine Girard, Mireille Ledevin, Cyrielle Lebreton, Agnès Hivonnait, Anna Creismeas, Marine Allais, Basile Marie, Justine Guguin, Véronique Blouin, Séverine Remy, Ignacio Anegon, Corinne Huchet, Alberto MalerbaBetty Kao, Anita Le Heron, Philippe Moullier, George Dickson, Linda Popplewell, Oumeya Adjali, Federica Montanaro, Caroline Le Guiner

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Original language	English
Pages (from-to)	520-535
Number of pages	16
Journal	Gene Therapy
Volume	29
Early online date	1 Feb 2022
DOIs	https://doi.org/10.1038/s41434-022-00317-6
Publication status	E-pub ahead of print - 1 Feb 2022

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10.1038/s41434-022-00317-6

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Bourdon, A., François, V., Zhang, L., Lafoux, A., Fraysse, B., Toumaniantz, G., Larcher, T., Girard, T., Ledevin, M., Lebreton, C., Hivonnait, A., Creismeas, A., Allais, M., Marie, B., Guguin, J., Blouin, V., Remy, S., Anegon, I., Huchet, C., ... Le Guiner, C. (2022). Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model. Gene Therapy, 29, 520-535. Advance online publication. https://doi.org/10.1038/s41434-022-00317-6

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title = "Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model",

author = "Audrey Bourdon and Virginie Fran{\c c}ois and Liwen Zhang and Aude Lafoux and Bodvael Fraysse and Gilles Toumaniantz and Thibaut Larcher and Tiphaine Girard and Mireille Ledevin and Cyrielle Lebreton and Agn{\`e}s Hivonnait and Anna Creismeas and Marine Allais and Basile Marie and Justine Guguin and V{\'e}ronique Blouin and S{\'e}verine Remy and Ignacio Anegon and Corinne Huchet and Alberto Malerba and Betty Kao and {Le Heron}, Anita and Philippe Moullier and George Dickson and Linda Popplewell and Oumeya Adjali and Federica Montanaro and {Le Guiner}, Caroline",

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Bourdon, A, François, V, Zhang, L, Lafoux, A, Fraysse, B, Toumaniantz, G, Larcher, T, Girard, T, Ledevin, M, Lebreton, C, Hivonnait, A, Creismeas, A, Allais, M, Marie, B, Guguin, J, Blouin, V, Remy, S, Anegon, I, Huchet, C, Malerba, A, Kao, B, Le Heron, A, Moullier, P, Dickson, G, Popplewell, L, Adjali, O, Montanaro, F & Le Guiner, C 2022, 'Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model', Gene Therapy, vol. 29, pp. 520-535. https://doi.org/10.1038/s41434-022-00317-6

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T1 - Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model

AU - Bourdon, Audrey

AU - François, Virginie

AU - Zhang, Liwen

AU - Lafoux, Aude

AU - Fraysse, Bodvael

AU - Toumaniantz, Gilles

AU - Larcher, Thibaut

AU - Girard, Tiphaine

AU - Ledevin, Mireille

AU - Lebreton, Cyrielle

AU - Hivonnait, Agnès

AU - Creismeas, Anna

AU - Allais, Marine

AU - Marie, Basile

AU - Guguin, Justine

AU - Blouin, Véronique

AU - Remy, Séverine

AU - Anegon, Ignacio

AU - Huchet, Corinne

AU - Malerba, Alberto

AU - Kao, Betty

AU - Le Heron, Anita

AU - Moullier, Philippe

AU - Dickson, George

AU - Popplewell, Linda

AU - Adjali, Oumeya

AU - Montanaro, Federica

AU - Le Guiner, Caroline

PY - 2022/2/1

Y1 - 2022/2/1

U2 - 10.1038/s41434-022-00317-6

DO - 10.1038/s41434-022-00317-6

M3 - Article

C2 - 35105949

SN - 0969-7128

VL - 29

SP - 520

EP - 535

JO - Gene Therapy

JF - Gene Therapy

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