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
. 2020 Sep 20:19:236-249.
doi: 10.1016/j.omtm.2020.09.009. eCollection 2020 Dec 11.

Adipose Tissue: An Emerging Target for Adeno-associated Viral Vectors

Affiliations
Review

Adipose Tissue: An Emerging Target for Adeno-associated Viral Vectors

Rhiannon Bates et al. Mol Ther Methods Clin Dev. .

Abstract

Adipose tissue is one of the largest organs, playing important roles in physiology and pathologies of multiple diseases. However, research related to adeno-associated virus (AAV) targeting adipose tissue has been left far behind studies carried out in the liver, brain, heart, and muscle. Despite initial reports indicating poor performance, AAV-mediated gene delivery to adipose tissue has continued to rise during the past two decades. AAV8 and a novel engineered hybrid serotype, Rec2, have been shown to transduce adipose tissue more efficiently than other serotypes so far tested and have been applied in most of the in vivo studies. The Rec2 serotype displays high efficacy of gene transfer to both brown and white fat via local and systemic administration. This review summarizes the advances in developing AAV vectors with enhanced adipose tropism and restricting off-target transgene expression. We discuss the challenges and strategies to search for and generate novel serotypes with tropism tailoring for adipose tissue and develop AAV vector systems to improve adipose transgene expression for basic research and translational studies.

Keywords: AAV; adipose; capsid engineering; gene therapy; tissue tropism.

PubMed Disclaimer

Figures

None
Graphical abstract
Figure 1
Figure 1
Components of the AAV Vector Targeting Adipose Tissue

Similar articles

Cited by

References

    1. Colella P., Ronzitti G., Mingozzi F. Emerging issues in AAV-mediated in vivo gene therapy. Mol. Ther. Methods Clin. Dev. 2017;8:87–104. - PMC - PubMed
    1. Sonntag F., Köther K., Schmidt K., Weghofer M., Raupp C., Nieto K., Kuck A., Gerlach B., Böttcher B., Müller O.J. The assembly-activating protein promotes capsid assembly of different adeno-associated virus serotypes. J. Virol. 2011;85:12686–12697. - PMC - PubMed
    1. Earley L.F., Powers J.M., Adachi K., Baumgart J.T., Meyer N.L., Xie Q., Chapman M.S., Nakai H. Adeno-associated virus (AAV) assembly-activating protein is not an essential requirement for capsid assembly of AAV serotypes 4, 5, and 11. J. Virol. 2017;91 e01980-16. - PMC - PubMed
    1. Chejanovsky N., Carter B.J. Mutagenesis of an AUG codon in the adeno-associated virus rep gene: effects on viral DNA replication. Virology. 1989;173:120–128. - PubMed
    1. King J.A., Dubielzig R., Grimm D., Kleinschmidt J.A. DNA helicase-mediated packaging of adeno-associated virus type 2 genomes into preformed capsids. EMBO J. 2001;20:3282–3291. - PMC - PubMed