Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A

Boutary et al. describe siRNA based therapy conjugated with squalene nanoparticles as an efficient approach to normalize PMP22 protein levels, restore locomotor activity, electrophysiological parameters and function of myelin sheath in CMT1A mouse models. These findings could be useful to develop th...

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Autores principales:	Suzan Boutary, Marie Caillaud, Mévidette El Madani, Jean-Michel Vallat, Julien Loisel-Duwattez, Alice Rouyer, Laurence Richard, Céline Gracia, Giorgia Urbinati, Didier Desmaële, Andoni Echaniz-Laguna, David Adams, Patrick Couvreur, Michael Schumacher, Charbel Massaad, Liliane Massaad-Massade
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Biology (General) QH301-705.5
Acceso en línea:	https://doaj.org/article/1040014b286640ea9f55ad4f4445c8c9
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spelling	oai:doaj.org-article:1040014b286640ea9f55ad4f4445c8c92021-12-02T13:34:50ZSqualenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A10.1038/s42003-021-01839-22399-3642https://doaj.org/article/1040014b286640ea9f55ad4f4445c8c92021-03-01T00:00:00Zhttps://doi.org/10.1038/s42003-021-01839-2https://doaj.org/toc/2399-3642Boutary et al. describe siRNA based therapy conjugated with squalene nanoparticles as an efficient approach to normalize PMP22 protein levels, restore locomotor activity, electrophysiological parameters and function of myelin sheath in CMT1A mouse models. These findings could be useful to develop therapeutic strategies for inherited peripheral neuropathies.Suzan BoutaryMarie CaillaudMévidette El MadaniJean-Michel VallatJulien Loisel-DuwattezAlice RouyerLaurence RichardCéline GraciaGiorgia UrbinatiDidier DesmaëleAndoni Echaniz-LagunaDavid AdamsPatrick CouvreurMichael SchumacherCharbel MassaadLiliane Massaad-MassadeNature PortfolioarticleBiology (General)QH301-705.5ENCommunications Biology, Vol 4, Iss 1, Pp 1-14 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Biology (General) QH301-705.5
spellingShingle	Biology (General) QH301-705.5 Suzan Boutary Marie Caillaud Mévidette El Madani Jean-Michel Vallat Julien Loisel-Duwattez Alice Rouyer Laurence Richard Céline Gracia Giorgia Urbinati Didier Desmaële Andoni Echaniz-Laguna David Adams Patrick Couvreur Michael Schumacher Charbel Massaad Liliane Massaad-Massade Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A
description	Boutary et al. describe siRNA based therapy conjugated with squalene nanoparticles as an efficient approach to normalize PMP22 protein levels, restore locomotor activity, electrophysiological parameters and function of myelin sheath in CMT1A mouse models. These findings could be useful to develop therapeutic strategies for inherited peripheral neuropathies.
format	article
author	Suzan Boutary Marie Caillaud Mévidette El Madani Jean-Michel Vallat Julien Loisel-Duwattez Alice Rouyer Laurence Richard Céline Gracia Giorgia Urbinati Didier Desmaële Andoni Echaniz-Laguna David Adams Patrick Couvreur Michael Schumacher Charbel Massaad Liliane Massaad-Massade
author_facet	Suzan Boutary Marie Caillaud Mévidette El Madani Jean-Michel Vallat Julien Loisel-Duwattez Alice Rouyer Laurence Richard Céline Gracia Giorgia Urbinati Didier Desmaële Andoni Echaniz-Laguna David Adams Patrick Couvreur Michael Schumacher Charbel Massaad Liliane Massaad-Massade
author_sort	Suzan Boutary
title	Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A
title_short	Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A
title_full	Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A
title_fullStr	Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A
title_full_unstemmed	Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A
title_sort	squalenoyl sirna pmp22 nanoparticles are effective in treating mouse models of charcot-marie-tooth disease type 1 a
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/1040014b286640ea9f55ad4f4445c8c9
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Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A

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