Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2018 May 21;6(2):42.
doi: 10.3390/diseases6020042.

Viral Vectors in Gene Therapy

Kenneth Lundstrom  1
Affiliations
Review

Viral Vectors in Gene Therapy

Kenneth Lundstrom. Diseases. .

Abstract

Applications of viral vectors have found an encouraging new beginning in gene therapy in recent years. Significant improvements in vector engineering, delivery, and safety have placed viral vector-based therapy at the forefront of modern medicine. Viral vectors have been employed for the treatment of various diseases such as metabolic, cardiovascular, muscular, hematologic, ophthalmologic, and infectious diseases and different types of cancer. Recent development in the area of immunotherapy has provided both preventive and therapeutic approaches. Furthermore, gene silencing generating a reversible effect has become an interesting alternative, and is well-suited for delivery by viral vectors. A number of preclinical studies have demonstrated therapeutic and prophylactic efficacy in animal models and furthermore in clinical trials. Several viral vector-based drugs have also been globally approved.

Keywords: approved drugs; clinical trials; gene silencing; immunotherapy; prevention; therapy.

PubMed Disclaimer

Conflict of interest statement

The authors declare no conflict of interest.

References

    1. Raper S.E., Chirmule N., Lee F.S., Wivel S.A., Bagg A., Gao G.P., Wilson J.M., Batshaw M.L. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol. Genet. Metab. 2003;80:148–158. doi: 10.1016/j.ymgme.2003.08.016. - DOI - PubMed
    1. McCormack M.P., Rabbitts T.H. Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 2004;350:913–922. doi: 10.1056/NEJMra032207. - DOI - PubMed
    1. Hacein-Bey-Abina S., Garrigue A., Wang G.P., Soulier J., Lim A., Morillon E., Clappier E., Caccavelli L., Delabesse E., Beldjord K., et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Investig. 2008;118:3132–3142. doi: 10.1172/JCI35700. - DOI - PMC - PubMed
    1. Schiedner G., Morral N., Parks R.S., Wu Y., Koopmans S.C., Langston C., Graham F.L., Beaudet A.L., Kochanek S. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat. Genet. 1998;18:180–183. doi: 10.1038/ng0298-180. - DOI - PubMed
    1. Wang F., Wang Z., Tian H., Qi M., Zhai Z., Li S., Li R., Zhang H., Wang W., Fu S., et al. Biodistribution and safety assessment of bladder cancer specific oncolytic adenovirus in subcutaneous xenografts tumor model in nude mice. Curr. Gene Ther. 2012;12:67–76. doi: 10.2174/156652312800099599. - DOI - PMC - PubMed