Adeno-associated virus (AAV) vectors have been widely adopted for use in gene therapy. A new study raises concerns regarding this approach, reporting that chromosomal insertions of AAV serotype 2 seem to activate proto-oncogenes in human hepatocellular carcinoma.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$209.00 per year
only $17.42 per issue
Rent or buy this article
Prices vary by article type
from$1.95
to$39.95
Prices may be subject to local taxes which are calculated during checkout
References
Njei, B., Rotman, Y., Ditah, I. & Lim, J.K. Hepatology 61, 191–199 (2015).
Sung, W.K. et al. Nat. Genet. 44, 765–769 (2012).
Paterlini-Bréchot, P. et al. Oncogene 22, 3911–3916 (2003).
Nault, J.-C. et al. Nat. Genet. 47, 1187–1193 (2015).
Weitzman, M.D. & Linden, R.M. Methods Mol. Biol. 807, 1–23 (2011).
Haberman, R.P., McCown, T.J. & Samulski, R.J. J. Virol. 74, 8732–8739 (2000).
Riordan, J.D. et al. PLoS Genet. 9, e1003441 (2013).
Donsante, A. et al. Science 317, 477 (2007).
Ranzani, M. et al. Nat. Methods 10, 155–161 (2013).
Wang, P.R. et al. Proc. Natl. Acad. Sci. USA 109, 11264–11269 (2012).
Chandler, R.J. et al. J. Clin. Invest. 125, 870–880 (2015).
Barzel, A. et al. Nature 517, 360–364 (2015).
Jiang, C.M. et al. World J. Gastroenterol. 20, 16464–16473 (2014).
Russell, D.W., Miller, A.D. & Alexander, I.E. Proc. Natl. Acad. Sci. USA 91, 8915–8919 (1994).
Author information
Authors and Affiliations
Corresponding authors
Ethics declarations
Competing interests
Scientific Advisory Board, Horizon Discovery. Equity in Universal Cells, Inc.
Rights and permissions
About this article
Cite this article
Russell, D., Grompe, M. Adeno-associated virus finds its disease. Nat Genet 47, 1104–1105 (2015). https://doi.org/10.1038/ng.3407
Published:
Issue Date:
DOI: https://doi.org/10.1038/ng.3407
This article is cited by
-
Cell and gene therapy for kidney disease
Nature Reviews Nephrology (2023)
-
Improving Molecular Therapy in the Kidney
Molecular Diagnosis & Therapy (2020)
-
Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome
Nature Genetics (2017)
-
Genetic basis of hepatitis virus-associated hepatocellular carcinoma: linkage between infection, inflammation, and tumorigenesis
Journal of Gastroenterology (2017)
-
Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?
Molecular Therapy (2016)