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

Complete list of metadata

https://hal-univ-evry.archives-ouvertes.fr/hal-02177575
Contributor : Evelyne Gicquel Connect in order to contact the contributor
Submitted on : Tuesday, July 9, 2019 - 10:57:12 AM
Last modification on : Wednesday, November 17, 2021 - 12:33:16 PM

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

Links full text

Identifiers

Collections

Citation

Export

Metrics

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

Links full text

Identifiers

Collections

Citation

Export

Share

Metrics