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AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression

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https://hal-univ-evry.archives-ouvertes.fr/hal-02177575
Contributor : Evelyne Gicquel <>
Submitted on : Tuesday, July 9, 2019 - 10:57:12 AM
Last modification on : Friday, September 18, 2020 - 2:34:59 PM

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Evelyne Gicquel, Natacha Maizonnier, Steven Foltz, William Martin, Nathalie Bourg, et al.. AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression. Human Molecular Genetics, Oxford University Press (OUP), 2017, 26 (10), pp.1952-1965. ⟨10.1093/hmg/ddx066⟩. ⟨hal-02177575⟩

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