Nature Communications (Apr 2021)
AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A
- Benoit Gautier,
- Helene Hajjar,
- Sylvia Soares,
- Jade Berthelot,
- Marie Deck,
- Scarlette Abbou,
- Graham Campbell,
- Maria Ceprian,
- Sergio Gonzalez,
- Claire-Maëlle Fovet,
- Vlad Schütza,
- Antoine Jouvenel,
- Cyril Rivat,
- Michel Zerah,
- Virginie François,
- Caroline Le Guiner,
- Patrick Aubourg,
- Robert Fledrich,
- Nicolas Tricaud
Affiliations
- Benoit Gautier
- INM, Univ. Montpellier, INSERM
- Helene Hajjar
- INM, Univ. Montpellier, INSERM
- Sylvia Soares
- Sorbonne Université, CNRS, INSERM, IBPS, Neuroscience Paris Seine
- Jade Berthelot
- INM, Univ. Montpellier, INSERM
- Marie Deck
- INM, Univ. Montpellier, INSERM
- Scarlette Abbou
- INM, Univ. Montpellier, INSERM
- Graham Campbell
- INM, Univ. Montpellier, INSERM
- Maria Ceprian
- INM, Univ. Montpellier, INSERM
- Sergio Gonzalez
- INM, Univ. Montpellier, INSERM
- Claire-Maëlle Fovet
- INSERM U1184, Immunology of Viral, Auto-immune, Hematological and Bacterial Diseases (ImVA-HB), IDMIT Department, CEA
- Vlad Schütza
- Institute of Anatomy, Leipzig University
- Antoine Jouvenel
- INM, Univ. Montpellier, INSERM
- Cyril Rivat
- INM, Univ. Montpellier, INSERM
- Michel Zerah
- Paediatric Neurosurgery Department, Université Paris Descartes and Assistance Publique-Hôpitaux de Paris, Hôpital Universitaire Necker
- Virginie François
- INSERM UMR 1089, Université de Nantes, CHU de Nantes
- Caroline Le Guiner
- INSERM UMR 1089, Université de Nantes, CHU de Nantes
- Patrick Aubourg
- Department of Paediatric Neurology, Centre Hospitalier Universitaire de Bicêtre
- Robert Fledrich
- Institute of Anatomy, Leipzig University
- Nicolas Tricaud
- INM, Univ. Montpellier, INSERM
- DOI
- https://doi.org/10.1038/s41467-021-22593-3
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 15
Abstract
Charcot-Marie-Tooth disease 1 A (CMT1A) results from PMP22 gene duplication and is characterized by peripheral nerve myelination deficits. Here, the authors prevent the development of pathological features in a rat model of CMT1A through the local delivery of AAV2/9 expressing shRNAs against PMP22.
