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Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A.
Boutary S, Caillaud M, El Madani M, Vallat JM, Loisel-Duwattez J, Rouyer A, Richard L, Gracia C, Urbinati G, Desmaële D, Echaniz-Laguna A, Adams D, Couvreur P, Schumacher M, Massaad C, Massaad-Massade L. Boutary S, et al. Among authors: loisel duwattez j. Commun Biol. 2021 Mar 9;4(1):317. doi: 10.1038/s42003-021-01839-2. Commun Biol. 2021. PMID: 33750896 Free PMC article.