AAV Joins the Rank of Genotoxic Vectors.
作者信息
Davé Utpal P, Cornetta Kenneth
机构信息
Division of Hematology/Oncology, Department of Medicine, Indiana University School of Medicine, Walther Hall, 980 W. Walnut St., Rm. C312J, Indianapolis, IN 46202, USA.
Department of Medical and Molecular Genetics, Indiana University School of Medicine, Walther Hall 980 W. Walnut Street, R3 C649, Indianapolis, IN 46202-5121, USA.
出版信息
Mol Ther. 2021 Feb 3;29(2):418-419. doi: 10.1016/j.ymthe.2021.01.007. Epub 2021 Jan 20.
Abstract
摘要
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Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease.增强针对脑的、基于 AAV 的组合疗法对克拉伯病的疗效和增加长期毒性。
Mol Ther. 2021 Feb 3;29(2):691-701. doi: 10.1016/j.ymthe.2020.12.031. Epub 2021 Jan 1.
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A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.一项对患有血友病 A 的狗进行的 AAV 基因治疗的长期研究发现，转导的肝细胞发生了克隆扩增。
Nat Biotechnol. 2021 Jan;39(1):47-55. doi: 10.1038/s41587-020-0741-7. Epub 2020 Nov 16.
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Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome.鉴定野生型 AAV2 基因组 3'非翻译区中的肝脏特异性增强子-启动子活性。
Nat Genet. 2017 Aug;49(8):1267-1273. doi: 10.1038/ng.3893. Epub 2017 Jun 19.
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