The Japanese Biochemical Society

[1P-327(2T16a-06)] Development of a more effective gene therapy drug for an inherited metabolic disease Galactosialidosis

Mizuki Miyoshi 1, Jun Tsukimoto1, Yuto Horii1, Yoshie Takeuchi2, Nijiho Kamori2, Rin Fukuike1, Michiko Kino3, Naozumi Ishimaru3, Kohji Itoh1,2 (1.Graduate School of Pharmaceutical Sciences, Tokushima University, 2.Faculty of Pharmacy, Tokushima University, 3.Graduate School of Oral Sciences, Tokushima University)

キーテクノロジー:遺伝子治療

先天代謝異常、アデノ随伴ウイルス、遺伝子治療、ミオクロニー発作、中枢神経症状

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