| Literature DB >> 30783966 |
Aman Patel1, Junling Zhao1, Dongsheng Duan1,2,3,4, Yi Lai5.
Abstract
Adeno-associated virus (AAV)-mediated gene therapy has evolved from bench to bedside, and now is the therapy of choice for certain inherited diseases. However, the small packaging capacity of AAV vectors prevents this technique from treating genetic diseases with mutations of large genes. Multiple strategies, including split AAV gene delivery and oversized AAV gene delivery, have been explored to deliver large gene expression cassettes. These strategies have gained some success in animal experiments. In this chapter, we review the progress of AAV-mediated delivery of large expression cassettes. We also review using AAV to deliver multiple transgenes.Entities:
Keywords: AAV; Dual vectors; Fragmented AAV gene delivery; Large gene expression cassette; Multiple gene expression cassette; Overlapping AAV vectors; Oversized AAV; Split AAV vectors; Trans-splicing AAV vectors; Triple vectors
Mesh：
Year: 2019 PMID： 30783966 DOI： 10.1007/978-1-4939-9139-6_2
Source DB: PubMed Journal: Methods Mol Biol ISSN： 1064-3745