AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models

Autor: Yoshiko Murakami, Saori Umeshita, Kae Imanishi, Yoshichika Yoshioka, Akinori Ninomiya, Takehiko Sunabori, Shibi Likhite, Masato Koike, Kathrin C. Meyer, Taroh Kinoshita

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101176- (2024)

Thirty genes are involved in the biosynthesis and modification of glycosylphosphatidylinositol (GPI)-anchored proteins, and defects in these genes cause inherited GPI deficiency (IGD). PIGA is X-linked and involved in the first step of GPI biosynthes

Externí odkaz: https://doaj.org/article/181bb1efa9434f12a9c25cb49690553b

AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models.

Autor: Murakami Y; Laboratory of Immunoglycobiology, Research Institute for Microbial Diseases, Osaka University, Suita, Osaka, Japan., Umeshita S; Laboratory of Immunoglycobiology, Research Institute for Microbial Diseases, Osaka University, Suita, Osaka, Japan., Imanishi K; Laboratory of Immunoglycobiology, Research Institute for Microbial Diseases, Osaka University, Suita, Osaka, Japan., Yoshioka Y; Graduate School of Frontier Bioscience, Osaka University, Suita, Osaka, Japan.; Center for Information and Neural Networks, National Institute of Information and Communications Technology (NICT), Osaka University, Suita, Osaka, Japan.; Center for Quantum Information and Quantum Biology, Osaka University, Suita, Osaka, Japan., Ninomiya A; Central Instrumentation Laboratory, Research Institute for Microbial Diseases, Osaka University, Suita, Osaka, Japan., Sunabori T; Department of Cell Biology and Neuroscience, Juntendo University Graduate School of Medicine, Bunkyo-ku, Tokyo, Japan., Likhite S; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, USA., Koike M; Department of Cell Biology and Neuroscience, Juntendo University Graduate School of Medicine, Bunkyo-ku, Tokyo, Japan., Meyer KC; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH, USA., Kinoshita T; Laboratory of Immunoglycobiology, Research Institute for Microbial Diseases, Osaka University, Suita, Osaka, Japan.; Center for Infectious Disease Education and Research, Osaka University, Suita, Osaka, Japan.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Dec 14; Vol. 32 (1), pp. 101176. Date of Electronic Publication: 2023 Dec 14 (Print Publication: 2024).