Last year's Nobel Prize in Physiology or Medicine was won by Andrew Fire and Craig Mello for their discovery of RNA interference, an evolutionarily conserved cellular pathway that regulates endogenous gene expression. In this Review, Gonzalez and Paulson discuss how RNA interference is being exploited experimentally to suppress the expression of dominantly acting genes that cause incurable neurodegenerative disorders, and highlight the questions that need to be answered before human clinical trials of this technology can commence.
- Pedro Gonzalez-Alegre
- Henry L Paulson