The Rise and Fall of RNAi
08/02/11 14:17 Filed in: GenomeWeb Daily Scan
Submitted by S. Pelech - Kinexus on Tue, 02/08/2011 - 14:17.
It took about two decades before the relatively simple process of how to produce monoclonal antibodies ultimately resulted in government-approved drugs for treatment of cancer and other diseases. However, monoclonal antibodies do not have to penetrate into target cells in order to exert their therapeutic effects. By contrast, RNAi's ideally must be delivered specifically inside of the cells that are diseased, and their unique mRNA targets must correspond to genes where there is a gain of function that is desirable to reduce. RNAi therapy would not be useful to combat the loss of specific tumour-suppressor gene functions, for example. It is intriguing that a lot more enthusiasm is growing for micro-RNA's, which appear to be much more potent for knocking out mRNA translation for protein synthesis. However, apart from the same delivery issues as RNAi's, micro-RNA's are rather non-specific and each may affect up to a hundred difference mRNA species. There is the potential to treat pathological productions of micro-RNA's with RNAi to mitigate their actions. This might ultimately prove to be the major utility for RNAi. Nevertheless, small molecule drugs remain the best option for molecular therapies, and based on historical data, it is going to be very hard to convince the pharmaceutical industry otherwise.
Link to the original blog post.
It took about two decades before the relatively simple process of how to produce monoclonal antibodies ultimately resulted in government-approved drugs for treatment of cancer and other diseases. However, monoclonal antibodies do not have to penetrate into target cells in order to exert their therapeutic effects. By contrast, RNAi's ideally must be delivered specifically inside of the cells that are diseased, and their unique mRNA targets must correspond to genes where there is a gain of function that is desirable to reduce. RNAi therapy would not be useful to combat the loss of specific tumour-suppressor gene functions, for example. It is intriguing that a lot more enthusiasm is growing for micro-RNA's, which appear to be much more potent for knocking out mRNA translation for protein synthesis. However, apart from the same delivery issues as RNAi's, micro-RNA's are rather non-specific and each may affect up to a hundred difference mRNA species. There is the potential to treat pathological productions of micro-RNA's with RNAi to mitigate their actions. This might ultimately prove to be the major utility for RNAi. Nevertheless, small molecule drugs remain the best option for molecular therapies, and based on historical data, it is going to be very hard to convince the pharmaceutical industry otherwise.
Link to the original blog post.