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The BBB, whilst vitally protecting the brain from harmful chemicals and infections, also makes the administration of medication to the brain problematic. Drugs are unable to cross the BBB and so, in order to reach the target for treatment, doctors are often forced to carry out invasive and painful procedures. However, this may all change following the impressive results of a recent landmark study.
The research, a joint collaboration between US and Korean scientists, published in the June 17 online edition of Nature , used a modified rabies virus peptide, termed CORVUS, to effectively deliver siRNA across the BBB in order to treat a viral infection in mice.
The researchers infected mice with Japanese encephalitis virus (JEV). Some of the mice then received intravenous administration of the antiviral siRNA bound to the rabies peptide. All of the mice infected with JEV who did not receive the injection died from the infection, compared with only 20% of the treated mice. Repeated administration of the therapy to mice did not trigger an inflammatory response, and no side effects of the treatment were found.
Lee Sang-kyung, coauthor of the study commented, "The RNA combined with the protein of a virus blocked the operation of the gene that begins the disease by getting into the blood cells. It is the first case to show that genetic substances to treat brain diseases can be delivered directly to the brain from blood vessels."
The authors are extremely hopeful that this new system of drug delivery will provide a noninvasive method of delivering siRNA, and possibly other therapeutic molecules, to the brain for the treatment of a wide variety of disorders, such as Alzheimer's disease and Parkinson's disease.
Source: Kumar P, Wu H, McBride JL et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature (2007) (Epub ahead of print).
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