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.

  • Research Highlight
  • Published:

Neurodevelopmental disorders

Miniaturized protein offers treatment hopes for Rett syndrome

Nature Reviews Neurology volume 13, page 708 (2017)Cite this article

Subjects

Access through your institution
Buy or subscribe

The restoration of two key domains in methyl-CpG-binding protein 2 (MECP2) could be sufficient to treat Rett syndrome, according to new research in mice. The study opens new avenues for development of a gene therapy for the condition.

Owing to the multitude of proteins bound by MECP2, characterization of the mechanisms that underlie Rett syndrome has been challenging. Symptoms were assumed to be irreversible as MECP2 mutations were thought to disrupt brain development; however, research in animal models has suggested that restoration of MECP2 function can ameliorate brain dysfunction and so could present a viable therapy.

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

Access options

Access through your institution

Buy this article

  • Purchase on SpringerLink
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

References

  1. Tillotson, R. et al. Radically truncated MeCP2 rescues Rett syndrome-like neurological defects. Nature 550, 398–401 (2017)

    Article  CAS  PubMed  PubMed Central  Google Scholar 

Download references

Authors
  1. Charlotte Ridler
    View author publications

    Search author on:PubMed Google Scholar

Related links

Related links

Related links in Nature Research

Leonard, H. et al. Clinical and biological progress over 50 years in Rett syndrome. Nat. Rev. Neurol. 13, 37–51 (2017)

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Ridler, C. Miniaturized protein offers treatment hopes for Rett syndrome. Nat Rev Neurol 13, 708 (2017). https://doi.org/10.1038/nrneurol.2017.156

Download citation

  • Published:

  • Issue date:

  • DOI: https://doi.org/10.1038/nrneurol.2017.156

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