[3S06a] Glycobiology leading to new drug modalities

1:22 PM - 1:41 PM JST(4:22 AM - 4:41 AM UTC)

[3S06a-02] Development of gene therapy for mucolipidosis with stem cell and gene editing

^○Tohru Sugawara¹, Ryunosuke Sanada^2,3, Akihiro Umezawa², Hidenori Akutsu^2,3, Yasuyuki Fukuhara⁴ (1.Graduate School of Medical Life Science, Yokohama City University , 2.Center for Regenerative Medicine, National Center for Child Health and Development, 3.Graduate School of Medical and Dental Sciences, Institute of Science Tokyo , 4.Division of Medical Genetics, National Center for Child Health and Development)

キーテクノロジー：iPS細胞とゲノム編集

Keywords:

ムコリピドーシス,ライソゾーム,GNPTAB,iPS細胞

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