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Adeno-associated viral vectors found free in media.

机译:在培养基中发现游离的腺相关病毒载体。

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摘要

Gene vectors derived from adeno-associated viruses (AAVs) are gaining strong momentum for in vivo applications, where efficient and long-term gene transfer is required and can be achieved by direct injection (Mueller and Flotte, 2008; Simonelli et at, 2010). Along with rapid expansion of the AAV toolbox, to which has been added novel natural serotypes and engineered capsids (Vandenberghe et al., 2009), the list of laboratories and publications using AAV is growing rapidly. Recombinant AAV has now become indisputably the most versatile in vivo gene delivery tool, not only for disease gene therapy but also for functional genomics and animal model development. Driven by the high demand, numerous new methods for large-scale vector production and purification have been made available (Zhang et ah, 2009). However, the devil is in the details. For researchers without prior experience and for studies that require large number of different vectors at a time, those details are still a significant issue.
机译:源自腺相关病毒 (AAV) 的基因载体在体内应用中获得了强劲的势头,其中需要高效和长期的基因转移,并且可以通过直接注射来实现(Mueller 和 Flotte,2008 年;Simonelli等人,2010)。随着AAV工具箱的快速扩展,增加了新的天然血清型和工程衣壳(Vandenberghe等人,2009),使用AAV的实验室和出版物的清单正在迅速增长。重组AAV现已无可争议地成为最通用的体内基因递送工具,不仅用于疾病基因治疗,还用于功能基因组学和动物模型开发。在高需求的推动下,已经提供了许多用于大规模载体生产和纯化的新方法(Zhang等人,2009)。然而,细节决定成败。对于没有经验的研究人员以及一次需要大量不同载体的研究来说,这些细节仍然是一个重大问题。

著录项

  • 来源
    《Human gene therapy》 |2010年第10期|1221-1222|共2页
  • 作者

    Xiao X;

  • 作者单位

    Division of Molecular Pharmaceutics, Eshelman School of Pharmacy, University of North Carolina at;

  • 收录信息
  • 原文格式 PDF
  • 正文语种 英语
  • 中图分类 治疗学;
  • 关键词

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