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Published online 26 March 2008 | Nature | doi:10.1038/news.2008.693
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Triumphs and tribulations for RNA interference
Two studies highlight promise and problems for gene silencing technique.
Researchers have managed to silence tiny chunks of RNA in monkeys using a gene-therapy technique. Their success could offer a new way to treat conditions from cancer to cardiovascular disease.
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If I recall correctly the AMD trials involve injecting naked RNAi which in my opinion would NOT necessarily enter the cells (where the RNAi would act). Therefore Ambati's results make sense. RNAi therapy suffers from the same problems as gene therapy i.e. not having an efficient delivery system
Recently great hype has been created regarding the therapeutic activity of the nucleotide molecules DNA &si RNA in almost all human diseases without following the basic principles of new drug development i.e., in vivo potency of the molecule, its toxicity, teratology and most important is the pharmacokinetic(ADME) aspects of the molecule and the cost of the synthesis of the nucleotide molecules. As Ambati rightly mentioned regarding the clinical activity of bevasiranib (si RNA) for AMD therapy, the experiments show the fortunate the activity of the molecule is due to positive immunogenic response of the nucleotide molecule rather than activity of the molecule itself, so it is clear one has to go for long term toxicity studies (3-4 years) for these nucleotide molecules before coming to the conclusion, their use in human therapeutics.