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IONIS-DNM2-2.5Rx (DYM101) is an antisense agent targeting dynamin 2. IONIS-DNM2-2.5Rx has the potential for the research of centronuclear myopathy (CNM)[1]. | [References]
[1] Tasfaout H, Buono S, Guo S, et al. Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice. Nat Commun. 2017;8:15661. DOI:10.1038/ncomms15661
[2] Buono S, Ross JA, Tasfaout H, et al. Reducing dynamin 2 (DNM2) rescues DNM2-related dominant centronuclear myopathy. Proc Natl Acad Sci U S A. 2018;115(43):11066-11071. DOI:10.1073/pnas.1808170115 |
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