Grieger Joshua C, Choi Vivian W, Samulski R Jude
Curriculum in Genetics and Molecular Biology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, USA.
Nat Protoc. 2006;1(3):1412-28. doi: 10.1038/nprot.2006.207.
The adeno-associated virus (AAV) is one of the most promising viral vectors for human gene therapy. As with any potential therapeutic system, a thorough understanding of it at the in vitro and in vivo levels is required. Over the years, numerous methods have been developed to better characterize AAV vectors. These methods have paved the way to a better understanding of the vector and, ultimately, its use in clinical applications. This review provides an up-to-date, detailed description of essential methods such as production, purification and titering and their application to characterize current AAV vectors for preclinical and clinical use.
腺相关病毒(AAV)是人类基因治疗中最有前景的病毒载体之一。与任何潜在的治疗系统一样,需要在体外和体内水平对其进行全面了解。多年来,已经开发出许多方法来更好地表征AAV载体。这些方法为更好地理解该载体并最终将其用于临床应用铺平了道路。本综述提供了诸如生产、纯化和滴度测定等基本方法的最新详细描述,以及它们在表征用于临床前和临床应用的当前AAV载体方面的应用。