Miniaturized protein offers treatment hopes for rett syndrome

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

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FAQs * Contact customer support REFERENCES * 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 * Charlotte Ridler View author publications You can also search for this author inPubMed 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 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: 27 October 2017 * Issue Date: December 2017 * DOI: https://doi.org/10.1038/nrneurol.2017.156 SHARE THIS ARTICLE

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