AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A

AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A

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....

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Autores principales: 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
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Publicado: Nature Portfolio 2021
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Acceso en línea:https://doaj.org/article/acd3f21814e64bcbb35c0a28138116c8
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spelling oai:doaj.org-article:acd3f21814e64bcbb35c0a28138116c82021-12-02T13:39:46ZAAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A10.1038/s41467-021-22593-32041-1723https://doaj.org/article/acd3f21814e64bcbb35c0a28138116c82021-04-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-22593-3https://doaj.org/toc/2041-1723Charcot-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.Benoit GautierHelene HajjarSylvia SoaresJade BerthelotMarie DeckScarlette AbbouGraham CampbellMaria CeprianSergio GonzalezClaire-Maëlle FovetVlad SchützaAntoine JouvenelCyril RivatMichel ZerahVirginie FrançoisCaroline Le GuinerPatrick AubourgRobert FledrichNicolas TricaudNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-15 (2021)
institution DOAJ
collection DOAJ
language EN
topic Science
Q
spellingShingle Science
Q
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
AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A
description 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.
format article
author 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
author_facet 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
author_sort Benoit Gautier
title AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A
title_short AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A
title_full AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A
title_fullStr AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A
title_full_unstemmed AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A
title_sort aav2/9-mediated silencing of pmp22 prevents the development of pathological features in a rat model of charcot-marie-tooth disease 1 a
publisher Nature Portfolio
publishDate 2021
url https://doaj.org/article/acd3f21814e64bcbb35c0a28138116c8
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