Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

Crossing the Rubicon

Nature Biotechnology volume 27, pages 42–44 (2009)Cite this article

A viral vector injected intravenously in mice crosses the blood-brain barrier and transduces widespread regions of the brain and spinal cord.

The gene therapy field has long awaited a viral vector that targets the central nervous system by crossing the blood-brain barrier (BBB). In a watershed paper in this issue, Kaspar and colleagues1 show that adeno-associated virus serotype 9 (AAV9) efficiently transduces brain cells located throughout the central nervous system in neonatal mice and, most surprisingly, in adult mice, after intravascular administration. They also provide substantial evidence that AAV9 does so by directly crossing the endothelial BBB. If these results can be reproduced in humans, they may facilitate gene therapy of diseases that affect large areas of the brain, such as motorneuron diseases, Parkinson's disease and Alzheimer's disease.

This is a preview of subscription content, access via your institution

Access options

Buy article

Get time limited or full article access on ReadCube.

$32.00

Buy

All prices are NET prices.

Figure 1: Developmental changes in the structure of the blood–brain barrier and the brain extracellular matrix likely influence the capacity of AAV9 to transduce different types of brain cells.

Kim Caesar

References

  1. Foust, K.D. et al. Nat. Biotechnol. 27, 59–65 (2008).

    Article  Google Scholar 

  2. Lowenstein, P.R. Trends Immunol. 23, 23–30 (2002).

    Article  CAS  Google Scholar 

  3. Bechmann, I. et al. Trends Immunol. 28, 5–11 (2007).

    Article  CAS  Google Scholar 

  4. Enquist, L.W. J. Infect. Dis. 186 Suppl 2, S209–14 (2002).

    Article  Google Scholar 

  5. Glorioso, J.C. & Fink, D.J. Mol. Ther. advance online publication, doi:10.1038/mt.2008.213 (7 October 2008).

  6. Fiandaca, M.S. et al. Neurotherapeutics 5, 123–127 (2008).

    Article  Google Scholar 

  7. Cearley, C.N. & Wolfe, J.H. J. Neurosci. 27, 9928–9940 (2007).

    Article  CAS  Google Scholar 

  8. Fu, H. et al. Mol. Ther. 8, 911–917 (2003).

    Article  CAS  Google Scholar 

  9. Di Pasquale, G. & Chiorini, J.A. Mol. Ther. 13, 506–516 (2006).

    Article  CAS  Google Scholar 

  10. Donsante, A. et al. Science 317, 477 (2007).

    Article  CAS  Google Scholar 

  11. Manno, C.S. et al. Nat. Med. 12, 342–347 (2006).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Pedro Lowenstein is at the Board of Governors' Gene Therapeutics Research Institute at the Cedars-Sinai Medical Center and the Department of Medicine and Pharmacology at UCLA, 8700 Beverly Blvd., Los Angeles, California 90049, USA. lowensteinp@cshs.org,

    Pedro R Lowenstein

Authors
  1. Pedro R Lowenstein
    View author publications

    You can also search for this author in PubMed Google Scholar

Rights and permissions

Reprints and Permissions

About this article

Cite this article

Lowenstein, P. Crossing the Rubicon. Nat Biotechnol 27, 42–44 (2009). https://doi.org/10.1038/nbt0109-42

Download citation

  • Issue Date:

  • DOI: https://doi.org/10.1038/nbt0109-42

This article is cited by

Search

Advanced search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing