Immortalized canine dystrophic myoblast cell lines for development of peptide-conjugated splice-switching oligonucleotides

Immortalized canine dystrophic myoblast cell lines for development of peptide-conjugated splice-switching oligonucleotides

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease caused by frameshift or nonsense mutations in the <i>DMD</i> gene, resulting in the loss of dystrophin from muscle membranes. Exon skipping using splice-switching oligonucleotides (SSOs) restores the reading frame of &l...

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Bibliografiska uppgifter
Huvudupphovsmän: Tone, Y, Mamchaoui, K, Tsoumpra, MK, Hashimoto, Y, Terada, R, Maruyama, R, Gait, MJ, Arzumanov, AA, McClorey, G, Imamura, M, Takeda, S, Yokota, T, Wood, MJA, Mouly, V, Aoki, Y
Materialtyp: Journal article
Språk:English
Publicerad: Mary Ann Liebert 2021

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