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）

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[O6-1] Identification of Genomic Instability Markers in Hematopoietic Stem Cells of Blmtm3Brd/tm3Brd Mice for Pre-clinical Trial of Lentiviral Gene Therapy to Prevent Hematological Malignancies in Bloom Syndrome Patients

^○Kazuhiro Noguchi, Yasuhiro Ikawa, Mika Takenaka, Yuta Sakai, Toshihiro Fujiki, Taizo Wada (Department of Pediatrics, Kanazawa University Hospital, Kanazawa, Japan)

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

^○Rin Fukuike^1,2, Jun Tsukimoto¹, Yuto Horii¹, Yoshie Takeuchi³, Nijiho Kamori³, Mizuki Miyoshi¹, Yasuo Shinohara^1,2, Kohji Itoh^1,4 (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)

[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)

[O6-4] Site-specific gene editing of mutated CD40L in T cells for treating the X-linked hyper-IgM syndrome

^○Akane Miura¹, Toru Uchiyama¹, Toru Yasuda¹, Kaori Edasawa¹, Yuriko Ishikawa², Masaki Yamada², Kazuhiko Nakabayashi³, Yukiko Ando¹, Kenichi Imadome², Masafumi Onodera¹ (1.Department of Human Genetics, National Center for Child Health and Development, 2.Division of Advanced Medicine for Virus Infections, National Center for Child Health and Development, 3.Department of Maternal-Fetal Biology, National Center for Child Health and Development)