Ribonucleic acid interference: the relevance of molecular targeting and therapeutics to neurosurgical practice.

In October 2006, Andrew Z. Fire (Stanford University School of Medicine) and Craig C. Mello (University of Massachusetts Medical School) were awarded the Nobel Prize in Physiology or Medicine 2006 for their discovery of RNA interference (RNAi)—gene silencing by doublestranded DNA (http://nobelprize.org/). We have compiled an overview of this new technology, addressing the basic concepts of ribonucleic acid (RNA) interference (RNAi), as well as several therapeutic applications for neurological disorders and its relevance for neurosurgeons. Encouraging results with RNAi-based therapies have been achieved in models of neurological disease, revealing the potential for translation to the treatment of neurodegenerative diseases, including movement disorders, brain tumors, and pain.5,31 The clinical translation of this technology will have a direct impact into neurosurgery practice, requiring neurosurgical intervention for effective targeted delivery to the central nervous system (CNS).