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News and Views
Nature Medicine 10, 775 - 776 (2004)
doi:10.1038/nm0804-775
RNAi quashes polyQ
Natasha J Caplen1
- Natasha J. Caplen is at the Gene Silencing Section, Office of Science and Technology Partnerships, Office of the Director, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20892, USA. e-mail: ncaplen@mail.nih.gov
Abstract
RNA interference in the brain inhibits neurodegeneration in a polyglutamine disease, SCA1. Is this now the way forward for the clinical treatment of certain genetic disorders (pages 816–820)?
RNA interference (RNAi), a post-transcriptional gene silencing mechanism first observed in plants and invertebrates, has become an invaluable tool for biologists studying gene function, particularly since its more recent identification in mammalian cells1, 2. The discovery that small, sequence-specific double-stranded RNAs could silence genes in mammals has also added new impetus to the development of nucleic acid–based therapeutic approaches to disease.
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