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

Evelyne Gicquel; Natacha Maizonnier; Steven Foltz; William Martin; Nathalie Bourg; Fedor Svinartchouk; Karine Charton; Aaron Beedle; Isabelle Richard

doi:10.1093/hmg/ddx066

Journal articles

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

Evelyne Gicquel ¹ Natacha Maizonnier ¹ Steven Foltz ² William Martin Nathalie Bourg ¹ Fedor Svinartchouk ¹ Karine Charton ¹ Aaron Beedle ² Isabelle Richard ¹

1 INTEGRARE - Approches génétiques intégrées et nouvelles thérapies pour les maladies rares

2 University of Georgia [USA]

Keywords : Muscle Dystrophy LGMD FKRP

Document type :

Journal articles

Domain :

Life Sciences [q-bio] / Human health and pathology

Life Sciences [q-bio] / Biotechnology

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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 : Thursday, March 4, 2021 - 3:50:24 PM

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

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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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