The 29th Annual Meeting of Japan Society of Gene and Cell Therapy

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Oral Session

[O6] Oral Session 6"Genetic Diseases"

Tue. Sep 12, 2023 10:10 AM - 10:50 AM Room 3 (1008, 10Ｆ, OICC)

Chairs:Yohta Shimada（Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine）,Toru Uchiyama（Division of Molecular Pathogenesis, Department of Human Genetics, National Center for Child Health and Development）

[O6-3] In vivo Gene therapy for lysosomal storage diseases mediated by AAV vector carrying BBB-penetrable enzyme

○Saki Matsushima1, Sayoko Iizuka1, Masafumi Kinoshita2, Haruna Takagi2, Shunsuke Iizuka2, Kota Ohtsuki2, Atsushi Imaikire2, Takashi Higuchi1, Yohta Shimada1, Hiroyuki Sonoda2, Toya Ohashi3, Hiroshi Kobayashi1 (1.Division of Gene Therapy, Research Center for Medical Sciences The Jikei University School of Medicine, 2.Research Division, JCR Pharmaceuticals, 3.The Jikei University of School of Nursing)

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