Gene therapy for NEU1 deficiencies by single intracerebroventricular administration using AAVPHP.eB carrying <em>modified NEU1</em> and <em>CTSA</em>

○Rin Fukuike

[26P2-am1-105S] Gene therapy for NEU1 deficiencies by single intracerebroventricular administration using AAVPHP.eB carrying modified NEU1 and CTSA

○Rin Fukuike¹, Jun Tsukimoto¹, Yuto Horii¹, Yoshie Takeuchi², Mizuki Miyoshi¹, Nijiho Kamori², Naozumi Ishimaru³, Noriko Kino³, Kohji Itoh^1,2 (1. Grad. Sch. Pharm. Sci., Tokushima Univ., 2. Facult. Pharm. Sci., Tokushima Univ., 3. Grad. Sch. Oral. Sci., Tokushima Univ.)

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Presentation information

[26P2-am1] Clinical pharmaceutical sciences/Others

[26P2-am1-105S] Gene therapy for NEU1 deficiencies by single intracerebroventricular administration using AAVPHP.eB carrying modified NEU1 and CTSA

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