Adeno-associated virus (AAV) vectors in cancer gene therapy

Jorge L Santiago-Ortiz; David V Schaffer

doi:10.1016/j.jconrel.2016.01.001

Adeno-associated virus (AAV) vectors in cancer gene therapy

J Control Release. 2016 Oct 28:240:287-301. doi: 10.1016/j.jconrel.2016.01.001. Epub 2016 Jan 12.

Authors

Jorge L Santiago-Ortiz¹, David V Schaffer²

Affiliations

¹ Department of Chemical and Biomolecular Engineering, University of California, Berkeley, CA, USA.
² Department of Chemical and Biomolecular Engineering, University of California, Berkeley, CA, USA; Department of Bioengineering, University of California, Berkeley, CA, USA; Department of Molecular and Cell Biology, University of California, Berkeley, CA, USA; The Helen Wills Neuroscience Institute, University of California, Berkeley, CA, USA. Electronic address: schaffer@berkeley.edu.

Abstract

Gene delivery vectors based on adeno-associated virus (AAV) have been utilized in a large number of gene therapy clinical trials, which have demonstrated their strong safety profile and increasingly their therapeutic efficacy for treating monogenic diseases. For cancer applications, AAV vectors have been harnessed for delivery of an extensive repertoire of transgenes to preclinical models and, more recently, clinical trials involving certain cancers. This review describes the applications of AAV vectors to cancer models and presents developments in vector engineering and payload design aimed at tailoring AAV vectors for transduction and treatment of cancer cells. We also discuss the current status of AAV clinical development in oncology and future directions for AAV in this field.

Keywords: AAV; Adeno-associated virus; Cancer; Gene delivery; Gene delivery vectors; Gene therapy.

Publication types

Review
Research Support, N.I.H., Extramural
Research Support, U.S. Gov't, Non-P.H.S.
Research Support, Non-U.S. Gov't

MeSH terms

Capsid Proteins / genetics
Clinical Trials as Topic
Dependovirus / genetics*
Gene Transfer Techniques*
Genetic Therapy / methods*
Genetic Vectors*
Humans
Neoplasms / genetics
Neoplasms / therapy*
Protein Engineering
Transgenes

Substances

Capsid Proteins

Grants and funding

R01 EY022975/EY/NEI NIH HHS/United States