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Wired reported yesterday on the fruits of a relatively new "drug" discovery processes that might lead to new migraine meds. What is most interesting about the story is the "drugs" in question, which aren't like drugs at all. The problem with most drugs, particularly in neuropharmacology, is that they are "dirty," as we call them - they bind to many biological targets, which can make their mechanisms of action difficult to understand. This technique sidesteps that difficulty in a very innovative fashion.
Proteins do the work of cells, which is to say absolutely everything you do, and their structures are what is being coded for within our so-called "genetic code" that lies along DNA strands. Genes are "read," and transcribed to an intermediary between DNA and protein known as "messenger" RNA. This mRNA takes the message to another region, where it is translated into a protein. Another kind of RNA - RNAi - was discovered fairly recently and the subject for which the Nobel Prize in Medicine was given to Mello & Fire in 2006. In theory, they are used by cells to shut down the mRNAs of one or many specific gene(s) so they are never translated, providing a potentially much more selective and rational approach to drug design compared to pharmacology.
The neatest thing that these guys did was their homework - trust me when I tell you that mRNA is a bitch to work with. In the body, it seems to be a very dynamically regulated process, and things called RNase can break the mRNA down quickly. RNase is everywhere outside of cells, and if you're not careful when doing work with mRNA, you will find it's all been eaten at the end of your experiment. This is why we convict killers on DNA rather than RNA evidence.
This company, however, took advantage of a strange property of molecules - stereoisometry - and designed mirror-image molecules of the sugar backbones of our RNA molecules. Think of it this way - your left hand is a mirror-image of your right hand. While they are identical, you can't make them overlap. Our bodies can't digest these mirror-image sugars, but they should still bind to their target mRNAs. Which, in theory, would make them a brilliant medium for drug discovery and could provide much-needed new insight into understanding human mental health and disorders thereof.
If I had money, I would probably invest in these guys, assuming they have stock available. They just got a pile of money from Eli-Lilly to do this, too...
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