Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.-论文-万方医学网

第一作者： Leszek,Lisowski

第一单位： 1] Stanford University, School of Medicine, Departments of Pediatrics and Genetics, 269 Campus Drive, Stanford, California 94305, USA [2] Gene Transfer, Targeting and Therapeutics Core, The Salk Institute for Biological Studies, 10010 N. Torrey Pines Rd, San Diego, California 92037, USA (L.L.); Department of Haematology, University College London Cancer Institute, London WC1E 6BT, UK (A.P.D.).

作者： Leszek,Lisowski ^[1] ; Allison P,Dane ^[2] ; Kirk,Chu ^[3] ; Yue,Zhang ^[3] ; Sharon C,Cunningham ^[4] ; Elizabeth M,Wilson ^[5] ; Sean,Nygaard ^[6] ; Markus,Grompe ^[6] ; Ian E,Alexander ^[7] ; Mark A,Kay ^[3]

作者单位： 1] Stanford University, School of Medicine, Departments of Pediatrics and Genetics, 269 Campus Drive, Stanford, California 94305, USA [2] Gene Transfer, Targeting and Therapeutics Core, The Salk Institute for Biological Studies, 10010 N. Torrey Pines Rd, San Diego, California 92037, USA (L.L.); Department of Haematology, University College London Cancer Institute, London WC1E 6BT, UK (A.P.D.). ^[1] 1] Gene Therapy Research Unit, The Children's Hospital at Westmead and Children's Medical Research Institute, Locked Bag 4001, Westmead, 2145 New South Wales, Australia [2] Gene Transfer, Targeting and Therapeutics Core, The Salk Institute for Biological Studies, 10010 N. Torrey Pines Rd, San Diego, California 92037, USA (L.L.); Department of Haematology, University College London Cancer Institute, London WC1E 6BT, UK (A.P.D.). ^[2] Stanford University, School of Medicine, Departments of Pediatrics and Genetics, 269 Campus Drive, Stanford, California 94305, USA. ^[3] Gene Therapy Research Unit, The Children's Hospital at Westmead and Children's Medical Research Institute, Locked Bag 4001, Westmead, 2145 New South Wales, Australia. ^[4] Yecuris Corporation, Portland, Oregon 97062, USA. ^[5] Oregon Stem Cell Center, Oregon Health and Science University, Portland, Oregon 97239, USA. ^[6] 1] Gene Therapy Research Unit, The Children's Hospital at Westmead and Children's Medical Research Institute, Locked Bag 4001, Westmead, 2145 New South Wales, Australia [2] Discipline of Paediatrics and Child Health, The University of Sydney, 2145 New South Wales, Australia. ^[7]

医学主题词动物(Animals);病毒壳体(Capsid);衣壳蛋白质类(Capsid Proteins);癌, 肝细胞(Carcinoma, Hepatocellular);细胞系, 肿瘤(Cell Line, Tumor);细胞, 培养的(Cells, Cultured);嵌合体(Chimera);临床试验(主题)(Clinical Trials as Topic);依赖病毒(Dependovirus);疾病模型, 动物(Disease Models, Animal);女(雌)性(Female);遗传载体(Genetic Vectors);肝细胞(Hepatocytes);人类(Humans);肝(Liver);男(雄)性(Male);小鼠(Mice);种特异性(Species Specificity);转导, 遗传(Transduction, Genetic);转基因(Transgenes)

DOI 10.1038/nature12875

PMID 24390344

发布时间 2024-05-22

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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.

Nature

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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.

Nature

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Nature

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