AAV-based gene therapy with modified HEXB confers lasting therapeutic benefits in GM2 gangliosidosis models.

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Title: AAV-based gene therapy with modified HEXB confers lasting therapeutic benefits in GM2 gangliosidosis models.
Authors: Kitakaze K; Department of Pharmacology, Kawasaki Medical School, 577 Matsushima, Kurashiki, Okayama 701-0192, Japan., Ohnishi Y; Department of Medicinal Biotechnology, Graduate School of Pharmaceutical Sciences, Tokushima University, 1-78-1 Sho-machi, Tokushima 770-8505, Japan., Tsuji D; Department of Medicinal Biotechnology, Graduate School of Pharmaceutical Sciences, Tokushima University, 1-78-1 Sho-machi, Tokushima 770-8505, Japan; Department of Medicinal Biotechnology, Faculty of Pharmaceutical Sciences, Tokushima University, 1-78-1 Sho-machi, Tokushima 770-8505, Japan., Watanabe R; Department of Medicinal Biotechnology, Graduate School of Pharmaceutical Sciences, Tokushima University, 1-78-1 Sho-machi, Tokushima 770-8505, Japan., Kamori N; Department of Medicinal Biotechnology, Faculty of Pharmaceutical Sciences, Tokushima University, 1-78-1 Sho-machi, Tokushima 770-8505, Japan., Katakai Y; The Corporation for Production and Research of Laboratory Primates, 1-16-2 Sakura, Tsukuba, Ibaraki 305-0003, Japan., Shibata H; The Corporation for Production and Research of Laboratory Primates, 1-16-2 Sakura, Tsukuba, Ibaraki 305-0003, Japan., Yoshizawa S; ONODERA GT Pharma, Inc., 3-25-22 Tonomachi, Kawasaki, Kanagawa 210-0821, Japan., Ito M; Division of Neurological Gene Therapy, Center for Open Innovation, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan., Takino N; Division of Neurological Gene Therapy, Center for Open Innovation, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan., Muramatsu SI; ONODERA GT Pharma, Inc., 3-25-22 Tonomachi, Kawasaki, Kanagawa 210-0821, Japan; Division of Neurological Gene Therapy, Center for Open Innovation, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan., Itoh K; Department of Medicinal Biotechnology, Graduate School of Pharmaceutical Sciences, Tokushima University, 1-78-1 Sho-machi, Tokushima 770-8505, Japan; Department of Medicinal Biotechnology, Faculty of Pharmaceutical Sciences, Tokushima University, 1-78-1 Sho-machi, Tokushima 770-8505, Japan; Division of Pediatrics, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan. Electronic address: kitoh@tokushima-u.ac.jp.
Source: Cell reports. Medicine [Cell Rep Med] 2026 May 19; Vol. 7 (5), pp. 102762. Date of Electronic Publication: 2026 Apr 22.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101766894 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2666-3791 (Electronic) Linking ISSN: 26663791 NLM ISO Abbreviation: Cell Rep Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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ISSN:2666-3791
DOI:10.1016/j.xcrm.2026.102762