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

Charcot-Marie-Tooth disease type 1 A (CMT1A) lacks an effective treatment. We provide a therapy for CMT1A, based on siRNA conjugated to squalene nanoparticles (siRNA PMP22-SQ NPs). Their administration resulted in normalization of Pmp22 protein levels, restored locomotor activity and electrophysiolo...

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Publicado en:Commun Biol
Autores principales: Boutary, Suzan, Caillaud, Marie, El Madani, Mévidette, Vallat, Jean-Michel, Loisel-Duwattez, Julien, Rouyer, Alice, Richard, Laurence, Gracia, Céline, Urbinati, Giorgia, Desmaële, Didier, Echaniz-Laguna, Andoni, Adams, David, Couvreur, Patrick, Schumacher, Michael, Massaad, Charbel, Massaad-Massade, Liliane
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group UK 2021
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7943818/
https://ncbi.nlm.nih.gov/pubmed/33750896
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s42003-021-01839-2
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