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RNA From Blood To Brain

  June, 27 2007 8:45
your information resource in human molecular genetics
It is now possible to perform gene therapy on the brain via intravenous injection, in mice at least. The finding, reported in Nature, opens a new potential line of noninvasive treatment for neuronal disease.

Getting any drug into the brain can be a problem because the blood-brain barrier acts as a barricade keeping systemic treatments out. Manjunath N. Swamy and colleagues show that when small fragments of RNA attached to a piece of viral protein are injected into the bloodstream of mice infected with a fatal form of encephalitis, the RNA-protein complex travels to the brain. The protein part binds neuronal cells, delivering the RNA part, which becomes internalised and can silence key genes through a process called RNA interference. The result - about 80% of treated animals survived, whilst all of the non-treated animals died.

Although the method needs tweaking to improve efficiency, the technique may offer a useful way of delivering nucleic acids and small-molecule drugs into the brain.

Author contact:

Manjunath N. Swamy (CBR Institute for Biomedical Research, Boston, MA, USA)
E-mail: swamy@cbrinstitute.org

(C) Nature press release.

Message posted by: Trevor M. D'Souza

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