Antisense-oligonucleotide-mediated-Dnm2-knockdown-prevents-and-reverts-myotubular-myopathy-in-mice-ncomms15661-s2

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Supplementary movie 1 Disease prevention through DNM2 targeting: phenotypes. (00:00 - 00:44) Comparison of 6 week-old Mtm1KO treated with 25 mg/kg of ASO ctrl and littermate Mtm1KO treated with 25 mg/kg of ASO#1.The Mtm1KO mouse treated with ASO ctrl was very affected with a pronounced kyphosis and inability to move, while the littermate Mtm1KO mouse injected with 25 mg/kg ASO#1 from 3 weeks of age does not display any particular clinical phenotype. (00:45 - 01:41) Effects of ASO#1 treatment at 11 weeks. 11 week-old Mtm1KO treated with 12.5 or 25 mg/kg of ASO#1 from 3 weeks of age presented a phenotype indistinguishable from wild-type mice.

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Tasfaout H, Buono S, Guo S, Kretz C, Messaddeq N, Booten S, Greenlee S, Monia B, Cowling B, Laporte J

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