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Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous system.

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第一作者： Jennifer L,Watts

第一单位： Steve and Cindy Rasmussen Institute for Genomic Medicine, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH., 43215, USA.

作者： Jennifer L,Watts ^[1] ; Shibi,Likhite ^[2] ; Yoshiko,Murakami ^[3] ; Taroh,Kinoshita ^[3] ; Kathrin,Meyer ^[4] ; Rolf W,Stottmann ^[5]

作者单位： Steve and Cindy Rasmussen Institute for Genomic Medicine, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH., 43215, USA. ^[1] Jerry R. Mendell Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH., 43215, USA. ^[2] Research Institute for Microbial Diseases, Osaka University, Suita, Osaka 565-0871 Japan. ^[3] Jerry R. Mendell Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH., 43215, USA.;Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH., 43210, USA. ^[4] Steve and Cindy Rasmussen Institute for Genomic Medicine, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH., 43215, USA.;Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH., 43210, USA. ^[5]

DOI 10.64898/2025.12.11.693709

PMID 41415370

发布时间 2025-12-29

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Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous system.

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Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous system.

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