Myotonic dystrophy

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  • 筋強直性ジストロフィー

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No effective treatment was available for myotonic dystrophy, even in animal model. We have established a new antisense oligonucleotide delivery to skeletal muscle of mice with bubble liposomes, and led to increased expression of chloride channel (CLCN1) protein and the amelioration of myotonia. In other experiments, we also identified small molecule compounds that correct aberrant splicing of Clcn1 gene. Manumycin A corrected aberrant splicing of Clcn1 in mouse model.

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  • Rinsho Shinkeigaku

    Rinsho Shinkeigaku 53 (11), 1109-1111, 2013

    Societas Neurologica Japonica

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