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Translational Pathway Toward a Clinical Trial Using the Second-Generation AAV Micro-Dystrophin Vector.

机译：使用第二代aaV微抗肌萎缩蛋白载体进行临床试验的转化途径。

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Duan, D.; Emter, C.; Lai, Y.; Yang, H. T.; Yue, Y.; Dai, A.;
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年度 2018
页码 1-328
总页数 328
原文格式 PDF
正文语种 eng
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入库时间 2022-08-29 10:46:01

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1. Targeted transgene integration into transgenic mouse fibroblasts carrying the full-length human AAVS1 locus mediated by HSV/AAV rep(+) hybrid amplicon vector. [J] . Bakowska JC, Di Maria MV, Camp SM, Gene therapy . 2003,第19期

机译：定向转基因整合到转基因小鼠成纤维细胞中，该小鼠成纤维细胞携带由HSV / AAV rep（+）杂交扩增子载体介导的全长人AAVS1基因座。
2. Packaging of an AAV vector encoding human acid alpha-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector. [J] . Sun B, Chen YT, Bird A, Molecular therapy: the journal of the American Society of Gene Therapy . 2003,第4期

机译：用改良的杂交腺病毒-AAV包装包装用于编码II型糖原贮积病的基因治疗的编码人酸性α-葡萄糖苷酶的AAV载体。
3. Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease [J] . Chady H. Hakim, Nathalie Clément, Lakmini P. Wasala, Molecular Therapy - Methods & Clinical Development . 2020,第3期

机译：通过瞬时转染和Herpesvirus系统制备的微染素AAV载体同样有效地治疗MDX小鼠肌肉疾病
4. Reverse Translational Research How Clinical Trials on Fluorescence Imaging for Vocal Cord Cancer Fuels Fundamental Research [C] . Olivier Gaiffe, Christian Pieralli, Laurent Tavernier, International Conference on Bio-inspired Systems and Signal Processing . 2013

机译：逆转转化研究如何对声带癌癌的荧光成像的临床试验促进基础研究
5. Sequential Rematched Randomization and Adaptive Monitoring with the Second-Generation p-Value to Increase the Efficiency and Efficacy of Randomized Clinical Trials [D] . Chipman, Jonathan Joseph. 2019

机译：用第二代P值进行顺序重复随机化和自适应监测，以提高随机临床试验的效率和功效
6. Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy [O] . Dongsheng Duan 2018

机译：系统性AAV微量肌营养不良蛋白基因治疗杜兴氏肌营养不良症
7. Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease [O] . Chady H. Hakim, Nathalie Clément, Lakmini P. Wasala, 2020

机译：通过瞬时转染和Herpesvirus系统制备的微染素AAV载体同样有效地治疗MDX小鼠肌肉疾病
8. Translational Pathway Toward a Clinical Trial Using the Second-Generation AAV Micro-Dystrophin Vector. [R] . Duan, D., Emter, C., Lai, Y., 2016

机译：使用第二代aaV微抗肌萎缩蛋白载体进行临床试验的转化途径。

Translational Pathway Toward a Clinical Trial Using the Second-Generation AAV Micro-Dystrophin Vector.

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