June 10, 2008 A new inhalable form of gene therapy -- based on technology recognized in the 2006 Nobel medicine prize, shows increasing promise for treating lung cancer, infectious diseases and inflammatory lung disease, scientists have concluded after an exhaustive review of worldwide research on the topic.
In the article, Sally-Ann Cryan, Niamh Durcan, and Charlotte Murphy focus on research efforts to develop an inhalable form of RNA interference (RNAi), a gene-therapy technique that interferes with or "silences" genes that make disease-causing proteins. The authors explain that RNAi has advantages over other gene therapies. It is potent, very specific, and appears to have a low risk of side effects.
They cite encouraging results with RNAi in laboratory studies in cells and animals with a range of lung diseases, including lung cancer, certain respiratory infections and inflammatory lung disease. Keys to successful therapy in humans include careful design of the gene-silencing agents, determining the most effective doses, and developing better ways of delivering RNAi agents to the lungs, the scientists say.
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- Durcan et al. Inhalable siRNA: Potential as a Therapeutic Agent in the Lungs. Molecular Pharmaceutics, 2008; 0 (0): 0 DOI: 10.1021/mp070048k
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