Characterization and biodistribution of under-employed gene therapy vector AAV7

未充分利用的基因治疗载体 AAV7 的表征和生物分布

阅读:9
作者:Samantha A Yost, Emre Firlar, Justin D Glenn, Hayley B Carroll, Steven Foltz, April R Giles, Jenny M Egley, Elad Firnberg, Sungyeon Cho, Trang Nguyen, William M Henry, Karolina J Janczura, Joseph Bruder, Ye Liu, Olivier Danos, Subha Karumuthil-Melethil, Sanjana Pannem, Valerie Yost, Yelena Engelson,

Abstract

The use of adeno-associated viruses (AAVs) as gene delivery vectors has vast potential for the treatment of many severe human diseases. Over one hundred naturally existing AAV capsid variants have been described and classified into phylogenetic clades based on their sequences. AAV8, AAV9, AAVrh.10, and other intensively studied capsids have been propelled into pre-clinical and clinical use, and more recently, marketed products; however, less-studied capsids may also have desirable properties (e.g., potency differences, tissue tropism, reduced immunogenicity, etc.) that have yet to be thoroughly described. These data will help build a broader structure-function knowledge base in the field, present capsid engineering opportunities, and enable the use of novel capsids with unique properties.

特别声明

1、本页面内容包含部分的内容是基于公开信息的合理引用;引用内容仅为补充信息,不代表本站立场。

2、若认为本页面引用内容涉及侵权,请及时与本站联系,我们将第一时间处理。

3、其他媒体/个人如需使用本页面原创内容,需注明“来源:[生知库]”并获得授权;使用引用内容的,需自行联系原作者获得许可。

4、投稿及合作请联系:info@biocloudy.com。