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Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS.

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第一作者： Tommaso,Iannitti

第一单位： University of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN), Department of Neuroscience, Sheffield, UK.

作者： Tommaso,Iannitti ^[1] ; Joseph M,Scarrott ^[1] ; Shibi,Likhite ^[2] ; Ian R P,Coldicott ^[1] ; Katherine E,Lewis ^[1] ; Paul R,Heath ^[1] ; Adrian,Higginbottom ^[1] ; Monika A,Myszczynska ^[1] ; Marta,Milo ^[1] ; Guillaume M,Hautbergue ^[1] ; Kathrin,Meyer ^[2] ; Brian K,Kaspar ^[3] ; Laura,Ferraiuolo ^[1] ; Pamela J,Shaw ^[4] ; Mimoun,Azzouz ^[5]

作者单位： University of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN), Department of Neuroscience, Sheffield, UK. ^[1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics, College of Medicine and Public Health, The Ohio State University, Columbus, OH, USA. ^[2] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics, College of Medicine and Public Health, The Ohio State University, Columbus, OH, USA; Department of Neuroscience, The Ohio State University, Columbus, OH, USA. ^[3] University of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN), Department of Neuroscience, Sheffield, UK. Electronic address: pamela.shaw@sheffield.ac.uk. ^[4] University of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN), Department of Neuroscience, Sheffield, UK. Electronic address: m.azzouz@sheffield.ac.uk. ^[5]

关键词 AAV9 ALS SOD1 biomarker cisterna magna clinical vector motor neuron neurodegeneration off-target effects shRNA

DOI 10.1016/j.omtn.2018.04.015

PMID 30195799

发布时间 2025-05-30

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