Research and Early Development, REGENXBIO Inc. , Rockville, Maryland, USA.
Institute of Quantitative Biomedicine and Rutgers CryoEM & Nanoimaging Facility, Rutgers, The State University of New Jersey , Piscataway, New Jersey, USA.
J Virol. 2023 Nov 30;97(11):e0116323. doi: 10.1128/jvi.01163-23. Epub 2023 Oct 16.
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.
腺相关病毒（AAV）作为基因传递载体，在治疗许多严重人类疾病方面具有巨大的潜力。已经描述了超过一百种天然存在的 AAV 衣壳变体，并根据其序列分为系统发育进化枝。AAV8、AAV9、AAVrh.10 和其他经过深入研究的衣壳已被推进临床前和临床使用，最近还有上市产品；然而，研究较少的衣壳也可能具有理想的特性（例如效力差异、组织嗜性、降低免疫原性等），这些特性尚未得到彻底描述。这些数据将有助于在该领域建立更广泛的结构-功能知识库，提供衣壳工程机会，并能够使用具有独特特性的新型衣壳。
