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.

Expanded CAG repeats in the crosshairs

Antisense oligomers targeted to CAG repeats allow allele-specific knockdown of the gene that causes Huntington's disease.

This is a preview of subscription content

Access options

Rent or Buy article

Get time limited or full article access on ReadCube.

from$8.99

All prices are NET prices.

Figure 1: Two nucleic acid-based strategies for allele-specific silencing of mutant genes containing expanded triplet repeats.

References

  1. 1

    Orr, H.T. & Zoghbi, H.Y. Annu. Rev. Neurosci. 30, 575–621 (2007).

    CAS  Article  Google Scholar 

  2. 2

    Hu, J. et al. Nat. Biotechnol. 27, 478–484 (2009).

    CAS  Article  Google Scholar 

  3. 3

    Snell, R.G. et al. Nat. Genet. 4, 393–397 (1993).

    CAS  Article  Google Scholar 

  4. 4

    Miller, V.M. et al. Proc. Natl. Acad. Sci. USA 100, 7195–7200 (2003).

    CAS  Article  Google Scholar 

  5. 5

    Schwarz, D.S. et al. PLoS Genet. 2, e140 (2006)

    Article  Google Scholar 

  6. 6

    van Bilsen, P.H. et al. Hum. Gene Ther. 19, 710–719 (2008).

    CAS  Article  Google Scholar 

  7. 7

    Zhang, Y. et al. J. Neurochem. 108, 82–90 (2009).

    CAS  Article  Google Scholar 

  8. 8

    Pfister, E.L. et al. Curr. Biol. published online, doi: 10.1016/j.cub.2009.003.030 (9 April 2009).

  9. 9

    Alves, S. et al. PLoS ONE 3, e3341 (2008).

    Article  Google Scholar 

Download references

Author information

Affiliations

Authors

Rights and permissions

Reprints and Permissions

About this article

Cite this article

Aronin, N. Expanded CAG repeats in the crosshairs. Nat Biotechnol 27, 451–452 (2009). https://doi.org/10.1038/nbt0509-451

Download citation

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