Department of Ophthalmology, University of Florida College of Medicine, Gainesville, FL, USA.
Methods Mol Biol. 2021;2225:77-92. doi: 10.1007/978-1-0716-1012-1_5.
Adeno-associated virus (AAV) is a helper-dependent single-stranded DNA parvovirus. Over the years, AAV has become the vector of choice in the gene therapy field due to its safety profile and low immunogenicity. With a carrying capacity of 4.2 kbp, these vectors have demonstrated their clinical value, especially in the field of ophthalmology. Herein we describe methods for the molecular design and packaging of AAV viral vectors. These methods apply to the design of single-stranded or self-complementary AAV vectors.
腺相关病毒（AAV）是一种依赖辅助病毒的单链 DNA 微小病毒。多年来，由于其安全性和低免疫原性，AAV 已成为基因治疗领域的首选载体。这些载体的载量为 4.2kbp，已显示出其临床价值，特别是在眼科领域。本文介绍了 AAV 病毒载体的分子设计和包装方法。这些方法适用于单链或自互补 AAV 载体的设计。
