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Allele-specific silencing therapy for Dynamin 2-related dominant centronuclear myopathy.
Trochet D, Prudhon B, Beuvin M, Peccate C, Lorain S, Julien L, Benkhelifa-Ziyyat S, Rabai A, Mamchaoui K, Ferry A, Laporte J, Guicheney P, Vassilopoulos S, Bitoun M. Trochet D, et al. Among authors: rabai a. EMBO Mol Med. 2018 Feb;10(2):239-253. doi: 10.15252/emmm.201707988. EMBO Mol Med. 2018. PMID: 29246969 Free PMC article.
Amphiphysin (BIN1) negatively regulates dynamin 2 for normal muscle maturation.
Cowling BS, Prokic I, Tasfaout H, Rabai A, Humbert F, Rinaldi B, Nicot AS, Kretz C, Friant S, Roux A, Laporte J. Cowling BS, et al. Among authors: rabai a. J Clin Invest. 2017 Dec 1;127(12):4477-4487. doi: 10.1172/JCI90542. Epub 2017 Nov 13. J Clin Invest. 2017. PMID: 29130937 Free PMC article.