Adeno-Associated Virus as Gene Delivery Vehicle into the Retina

  • Shuyun Deng
  • Kazuhiro OkaEmail author
Part of the Methods in Molecular Biology book series (MIMB, volume 2092)


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.

Key words

Adeno-associated virus Serotype Packaging Purification Titration 



We thank Dr. Sean Hartig for helpful discussion and critical reading of the manuscript. This work was supported by Gene Vector Core, Baylor College of Medicine Advanced Technology Cores.


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© Springer Science+Business Media, LLC, part of Springer Nature 2020

Authors and Affiliations

  1. 1.Advanced Technology CoresBaylor College of MedicineHoustonUSA
  2. 2.Department of Molecular and Cellular BiologyBaylor College of MedicineHoustonUSA

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