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腺相关病毒载体的生产、纯化和特性鉴定。

Production, purification and characterization of adeno-associated vectors.

机构信息

Center of Animal Biotechnology and Gene Therapy and Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, 08193-Bellaterra, Spain.

出版信息

Curr Gene Ther. 2010 Dec;10(6):423-36. doi: 10.2174/156652310793797685.

DOI:10.2174/156652310793797685
PMID:21054248
Abstract

The use of recombinant adeno-associated viral vectors (rAAVs) as gene transfer tools has increased dramatically during the past last several years, establishing AAV as the vector of choice for many therapeutic applications. With the steady advance of the field toward clinical studies, and the isolation and engineering of several novel AAV serotypes, efficient, scalable, and versatile production and purification methods are continuously under development. Here, we review the current state of the art in the various production and purification methods for rAAVs. Classical parameters and methodologies to characterize rAAV stocks will be also discussed.

摘要

在过去的几年中,重组腺相关病毒载体(rAAV)作为基因转移工具的使用急剧增加,使 AAV 成为许多治疗应用的首选载体。随着该领域向临床研究的稳步推进,以及几种新型 AAV 血清型的分离和工程化,高效、可扩展和多功能的生产和纯化方法不断得到开发。本文综述了 rAAV 的各种生产和纯化方法的最新进展。本文还将讨论 rAAV 库存的经典参数和方法学。

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