Protocol for Efficient Generation and Characterization of Adeno-Associated Viral Vectors.

Q1 Immunology and Microbiology

Human Gene Therapy Methods Pub Date : 2017-10-01 DOI:10.1089/hgtb.2017.192

Andreas Jungmann, Barbara Leuchs, Jean Rommelaere, Hugo A Katus, Oliver J Müller

引用次数: 38

Abstract

Adeno-associated virus vectors are a powerful tool for gene transfer approaches. We have established a simple and fast plasmid-based production system for achieving high adeno-associated virus titers within 6 working days. The same procedure can be used for all serotypes and thus allows direct comparability of different serotypes. In this protocol we describe a step-by-step procedure that results in well-characterized vectors suitable for both in vitro approaches and preclinical studies.

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高效生成和表征腺相关病毒载体的方法。

腺相关病毒载体是基因转移方法的有力工具。我们已经建立了一个简单快速的质粒生产系统，可以在6个工作日内获得高滴度的腺相关病毒。相同的程序可用于所有血清型，从而允许不同血清型的直接可比性。在本协议中，我们描述了一个循序渐进的过程，结果在体外方法和临床前研究中具有良好特征的载体。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Human Gene Therapy Methods BIOTECHNOLOGY & APPLIED MICROBIOLOGY-GENETICS & HEREDITY

CiteScore

5.80

自引率

0.00%

发文量

审稿时长

>12 weeks

期刊介绍： Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases. The Journal is divided into three parts. Human Gene Therapy, the flagship, is published 12 times per year. HGT Methods, a bimonthly journal, focuses on the applications of gene therapy to product testing and development. HGT Clinical Development, a quarterly journal, serves as a venue for publishing data relevant to the regulatory review and commercial development of cell and gene therapy products.