Engineering AAV receptor footprints for gene therapy.

Published

Journal Article (Review)

Adeno-associated viruses (AAV) are currently at the forefront of human gene therapy clinical trials as recombinant vectors. Significant progress has been made in elucidating the structure, biology and tropisms of different naturally occurring AAV isolates in the past decade. In particular, a spectrum of AAV capsid interactions with host receptors have been identified and characterized. These studies have enabled a better understanding of key determinants of AAV cell recognition and entry in different hosts. This knowledge is now being applied toward engineering new, lab-derived AAV capsids with favorable transduction profiles. The current review conveys a structural perspective of capsid-glycan interactions and provides a roadmap for generating synthetic strains by engineering AAV receptor footprints.

Full Text

Duke Authors

Cited Authors

  • Madigan, VJ; Asokan, A

Published Date

  • June 2, 2016

Published In

Volume / Issue

  • 18 /

Start / End Page

  • 89 - 96

PubMed ID

  • 27262111

Pubmed Central ID

  • 27262111

Electronic International Standard Serial Number (EISSN)

  • 1879-6265

International Standard Serial Number (ISSN)

  • 1879-6257

Digital Object Identifier (DOI)

  • 10.1016/j.coviro.2016.05.001

Language

  • eng