Adeno-Associated Virus as Gene Delivery Vehicle into the Retina

Shuyun Deng; Kazuhiro Oka

doi:10.1007/978-1-0716-0175-4_7

Adeno-Associated Virus as Gene Delivery Vehicle into the Retina

Methods Mol Biol. 2020:2092:77-90. doi: 10.1007/978-1-0716-0175-4_7.

Authors

Shuyun Deng¹, Kazuhiro Oka²

Affiliations

¹ Advanced Technology Cores, Baylor College of Medicine, Houston, TX, USA.
² Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, TX, USA. kazuhiro@bcm.edu.

PMID: 31786783
DOI: 10.1007/978-1-0716-0175-4_7

Abstract

Initially discovered as a contaminant of adenovirus preparations, adeno-associated virus (AAV) has proved one of the most promising viral vectors for human gene therapy. The safety profile of AAV has been well-characterized in vivo studies, and the first gene therapy for patients with vision loss caused by Leber congenital amaurosis or retinitis pigmentosa was approved by the US Food and Drug Administration in 2017. This is an exciting era for investigators working on retina biology and treatments for blindness. In this chapter, we provide detailed methods for laboratory-scale production, purification, and characterization of AAV.

Keywords: Adeno-associated virus; Packaging; Purification; Serotype; Titration.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Adenoviridae / genetics*
Cell Line
Eye Proteins / genetics
Gene Transfer Techniques
Genetic Therapy / methods
Genetic Vectors / genetics*
HEK293 Cells
Humans
Leber Congenital Amaurosis / genetics
Retina / physiology*
Retinitis Pigmentosa / genetics

Substances

Eye Proteins