First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.

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第一作者： Paul,Harmatz

第一单位： UCSF Benioff Children's Hospital Oakland, Oakland, CA 94609, USA. Electronic address: paul.harmatz@ucsf.edu.

作者： Paul,Harmatz ^[1] ; Carlos E,Prada ^[2] ; Barbara K,Burton ^[3] ; Heather,Lau ^[4] ; Craig M,Kessler ^[5] ; Liching,Cao ^[6] ; Marina,Falaleeva ^[6] ; Andres G,Villegas ^[6] ; Jennifer,Zeitler ^[6] ; Kathleen,Meyer ^[6] ; Weston,Miller ^[6] ; Cheryl,Wong Po Foo ^[6] ; Sagar,Vaidya ^[6] ; Wendy,Swenson ^[6] ; Lisa H,Shiue ^[6] ; Didier,Rouy ^[6] ; Joseph,Muenzer ^[7]

作者单位： UCSF Benioff Children's Hospital Oakland, Oakland, CA 94609, USA. Electronic address: paul.harmatz@ucsf.edu. ^[1] Division of Genetics, Birth Defects & Metabolism, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA; Department of Pediatrics, Feinberg School of Medicine of Northwestern University, Chicago, IL, USA; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. ^[2] Division of Genetics, Birth Defects & Metabolism, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA; Department of Pediatrics, Feinberg School of Medicine of Northwestern University, Chicago, IL, USA. ^[3] Department of Neurology, NYU School of Medicine, New York, NY, USA. ^[4] Georgetown University, Washington, DC, USA. ^[5] Sangamo Therapeutics, Inc., Brisbane, CA, USA. ^[6] University of North Carolina at Chapel Hill, Chapel Hill, NC, USA. ^[7]

关键词 MPS I MPS II first-in-human hemophilia in?vivo genome editing mucopolysaccharidosis zinc-finger nuclease

主题词人类(Humans)

DOI 10.1016/j.ymthe.2022.10.010

PMID 36299240

发布时间 2023-12-12

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