[3P-021（3T03a-09）] CDP-ribitol prodrug treatment ameliorates ISPD-deficient muscular dystrophy mouse model

^〇Hideki Tokuoka¹, Rieko Imae², Hitomi Nakashima¹, Hiroshi Manya², Chiaki Masuda³, Shunsuke Hoshino², Kazuhiro Kobayashi¹, Dirk J. Lefeber⁴, Riki Matsumoto¹, Takashi Okada⁵, Tamao Endo², Motoi Kanagawa^1,6, Tatsushi Toda⁷ (1.Division of Molecular Brain Science and Neurology, Kobe University Graduate School of Medicine, 2.Molecular Glycobiology, Research Team for Mechanism of Aging, Tokyo Metropolitan Geriatric Hospital and Institute of Gerontology, 3.Department of Biochemistry and Molecular Biology, Nippon Medical School, 4.Department of Neurology, Donders Institute for Brain, Cognition and Behavior, Radboud University Medical Center, 5.Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, 6.Department of Cell Biology and Molecular Medicine, Ehime University Graduate School of Medicine, 7.Department of Neurology, Graduate School of Medicine, The University of Tokyo)

キーテクノロジー：ドラッグデリバリーシステム

CDP-リビトール、ジストログリカン、プロドラッグ、筋ジストロフィー