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）

[O6-2] Gene therapy for NEU1 deficiencies using AAVPHP.eB carrying modified NEU1 and the human CTSA.

○Rin Fukuike1,2, Jun Tsukimoto1, Yuto Horii1, Yoshie Takeuchi3, Nijiho Kamori3, Mizuki Miyoshi1, Yasuo Shinohara1,2, Kohji Itoh1,4 (1.Graduate School of Pharmaceutical Sciences, Tokushima University, 2.Institute of Advanced Medical Sciences, 3.Faculty School of Pharmaceutical Sciences, Tokushima University, 4.Department of Pediatrics, Jichi Medical University School of Medicine)

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