New from NCP

16 July 2007

Therapeutic RNA interference in neurodegenerative disease

doi:10.1038/ncpneuro0551

In a review article in the July issue of Nature Clinical Practice Neurology, Pedro Gonzalez-Alegre and Henry L Paulson consider how RNA interference (RNAi) technology might be exploited to treat neurodegenerative diseases that are currently incurable, such as Huntington's disease, Alzheimer's disease and spinocerebellar ataxia.

The RNAi gene regulatory pathway could potentially suppress the expression of a wide range of disease-causing mutant genes. Initial studies of therapeutic RNAi in cell-based and animal models of neurodegenerative disease have produced encouraging results, and human trials are expected to be initiated within the next few years.

Andrew Fire and Craig Mello won the 2006 Nobel Prize in Physiology or Medicine for the discovery of the RNAi pathway. Since their landmark paper was published in 1998, RNAi has become a powerful tool for biological research, enabling virtually any target gene to be suppressed in a sequence-specific manner. In the current article, Gonzalez-Alegre and Paulson highlight the challenges involved in bringing RNAi to the neurology clinic, including safe and efficient delivery of the therapeutic RNAi agent, and the development of reliable measures of treatment efficacy.

Author contact:
Henry L Paulson (University of Michigan Medical School, Ann Arbor, MI, USA)
Tel: +1 319 335 8696; E-mail: henry-paulson@uiowa.edu

Editor contact:
Heather Wood (Editor, Nature Clinical Practice Neurology)
Tel: +44 20 7843 3609; E-mail: h.wood@nature.com

Media contacts:
Helen Jamison (Nature London)
Tel: +44 20 7843 4658; E-mail: h.jamison@nature.com

Ruth Francis (Nature London)
Tel: +44 20 7843 4562; E-mail: r.francis@nature.com

The Nature Clinical Practice journals
Nature Clinical Practice journals deliver timely, authoritative interpretations of key research developments, translating the latest findings into clinical practice. Each of the eight titles in the series is published monthly. Content includes editorial and opinion pieces, highlights from the current literature, commentaries on the application of recent research to practical patient care, thorough reviews, and in-depth case studies. The journals' Editors-in-Chief and international advisory boards ensure comprehensive coverage throughout the year, with discussion of topical issues included as soon as possible after publication of the original research.

The first four Nature Clinical Practice journals were launched in November 2004 and a further four were launched in November 2005. For a full list of journal titles and for more information, visit www.nature.com/clinicalpractice

About Nature Publishing Group
Nature Publishing Group (NPG) is a division of Macmillan Publishers Ltd, dedicated to serving the academic, professional scientific and medical communities. NPG's flagship title, Nature, was first published in 1869. Other publications include Nature research journals, Nature Reviews, Nature Clinical Practice, a range of prestigious academic journals, including society-owned publications. NPG also provides news content through news@nature.com and scientific career information through Naturejobs.

NPG is a global company, with headquarters in London and offices in New York, San Francisco, Washington DC, Boston, Tokyo, Paris, Munich, Hong Kong, Melbourne, Delhi, Mexico City and Basingstoke. For more information, please go to www.nature.com.

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