Editor's Summary
5 July 2007
Therapy with siRNAs
Since the discovery of gene silencing by naturally occurring small interfering RNA (siRNA) molecules, the idea that they might be used therapeutically has been up and running. Several reports of systemic delivery of siRNAs have been published, but the brain was not among the targets, because of that old problem, the blood–brain barrier. Now a collaboration between labs in the United States and South Korea has developed a way of delivering siRNAs across the barrier. The method, which is suitable for other types of drug as well as siRNA, makes use of a short peptide derived from the rabies virus as a transporter for the RNA. As well as delivering the RNA into neuronal cells in cell culture, an antiviral siRNA was delivered specifically into the brains of mice infected with encephalitis: about 80% of the mice survived the normally fatal infection. If replicated in humans, this work could lead to the development of noninvasive intravenous treatments for neurological disorders.
News and Views: Molecular medicine: Entry granted
The inability to efficiently deliver small interfering RNAs to target organs hinders their therapeutic application. So a demonstration of siRNA delivery to a notoriously difficult organ — the brain — is very exciting indeed.
Edouard M. Cantin & John J. Rossi
doi:10.1038/448033a
Article: Transvascular delivery of small interfering RNA to the central nervous system
Priti Kumar, Haoquan Wu, Jodi L. McBride, Kyeong-Eun Jung, Moon Hee Kim, Beverly L. Davidson, Sang Kyung Lee, Premlata Shankar & N. Manjunath
doi:10.1038/nature05901
Abstract | Full Text | PDF (841K) | Supplementary information
