RNA binding proteins: a new target for therapy?

In the ongoing effort to develop new therapy for diseases including cancer, identifying new drug targets in diseased cells remains the focus of much scientific research. In recent years, new potential drug targets and new molecules have been identified. These include proteins that are over-expressed or not expressed in disease cells compared with normal or gene variations that alter the shape or function of a protein in a cell. Examples of such are plentiful -- from overexpression of the protein Her2 in breast cancer, to the lack of expression of the tumor suppressor gene p53 in numerous cancer types.

In the last 10 years, new molecules called micro-RNA (which are small stretches of RNA molecules that can prevent proteins from being expressed) have stolen the spotlight and hold the potential to be developed into a new class of drugs that specifically target their function. This field is exploding and providing new insight into how genes and proteins are expressed. It now appears that the control of protein levels in a cell involves even more players.

Now, it seems that a newly scrutinized class of proteins, called RNA binding proteins, are emerging as a new target that can be exploited to develop new therapies. These RNA binding proteins are involved in RNA binding (couldn’t guess that from the name!). They are able to carry RNA to different locations in the cell, to increase or decrease the expression of RNA or protein, and, it now seems, have altered expression, function, or location in diseased cells including inflammation and cancer. RNA binding proteins can interact with miRNA to control the level of the bound RNA or the level of protein expression. Understanding their role in gene expression does add yet another layer to the complexity of the cell, but may lead to critical insight into how to control expression of important proteins.

Recent review articles and commentaries have shown the growing number of diseases that have altered RNA binding protein activity. Maybe they are an emerging new avenue for therapy? Check out these reviews:

http://onlinelibrary.wiley.com/doi/10.1002/wrna.62/abstract;jsessionid=1E2822BB213813E5BA84E8888B9F8AB9.d01t01


http://www.springerlink.com/content/35862187j2587526/


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miRNAs strike again!

The ever-increasing number of articles documenting how miRNAs are key players in gene regulation, where overexpression or underexpression of a myriad of miRNAs results in disease states, continues to rise. Clearly, gene regulation is complex and control of transcription or translation is a key determinate in the development of disease. MicroRNAs are proving to be critical mediators of disease states and could potentially be therapeutic targets. Combined with other forms of regulation (epigenetic, transcriptional regulation, post-transcriptional modifications, and so on...) it will be possible to understand how cells specially fine-tune gene expression to maximize performance and prevent disease. Once a complete picture -- involving miRNAs, proteins, RNA, DNA, epigenetic mediators -- is fully realized, then real targeted therapy can be designed. But first, we must understand how each component works. Every new paper documenting how miRNAs control genes and disease is another step to understanding how to successfully target diseases with pharmacologic agents.