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

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

doi:10.1038/s42003-021-01839-2

Communications Biology (Mar 2021)

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

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

Affiliations

Suzan Boutary: U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay
Marie Caillaud: U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay
Mévidette El Madani: U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay
Jean-Michel Vallat: Service de Neurologie - Centre de Référence Neuropathies Périphérique Rares, CHU de Limoges - Hôpital Dupuytren, 2 Avenue Martin Luther King
Julien Loisel-Duwattez: U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay
Alice Rouyer: U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay
Laurence Richard: Service de Neurologie - Centre de Référence Neuropathies Périphérique Rares, CHU de Limoges - Hôpital Dupuytren, 2 Avenue Martin Luther King
Céline Gracia: UMR 8203 CNRS, newly UMR 9018 CNRS, Université Paris-Saclay
Giorgia Urbinati: UMR 8203 CNRS, newly UMR 9018 CNRS, Université Paris-Saclay
Didier Desmaële: Institut Galien Paris-Sud, CNRS UMR 8612, Université Paris-Sud, Université Paris-Saclay
Andoni Echaniz-Laguna: U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay
David Adams: U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay
Patrick Couvreur: Institut Galien Paris-Sud, CNRS UMR 8612, Université Paris-Sud, Université Paris-Saclay
Michael Schumacher: U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay
Charbel Massaad: Faculty of Basic and Biomedical Sciences, Paris Descartes University, INSERM UMRS 1124
Liliane Massaad-Massade: U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay

DOI: https://doi.org/10.1038/s42003-021-01839-2
Journal volume & issue: Vol. 4, no. 1
pp. 1 – 14

Abstract

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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.

Published in Communications Biology

ISSN: 2399-3642 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science: Biology (General)
Website: https://www.nature.com/commsbio/

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