Fighting Disease at the Source

Our bodies are amazing machines, millions of processes and cycles working together keeping us alive. However as modern medicine learns more about these bodies, and consequently how to heal them, certain problems arise. It is now know that some conditions are genetic, coded into our very DNA. Ideas on how to treat these conditions vary and almost exclusively focus on controlling the results instead of focusing on the problem genes themselves. However recent medical developments may just change that.

There is a new approach to medicine design that works using molecules known as interference RNA (iRNA). These double stranded sections of RNA disrupt the normal protein synthesis process by targeting and destroying select mRNA strands, effectively preventing their translation into protein. With each iRNA strand able to destroy several thousand mRNA strands, this effectively silences the gene; with none of the mRNA being translated it’s like it wasn’t even there. Theoretically this process could be used to fight any disease or condition involving mRNA, and currently it is being developed to fight diseases involving the synthesis of proteins.

This is truly a wonderful new approach providing what could be a pardon to a genetic death sentence. However, whenever dealings with DNA in humans certain worries arise; questions about safety and ethics become paramount. While iRNA is designed to target specific mRNA strands, it is possible that some iRNA may cross-react with healthy or necessary mRNA strands with sequence similarity, possibly leading to healthy genes being unintentionally silenced. Thankfully this process isn’t getting rushed into production, but is going through rigorous clinical trials. Even recently studies found that large doses of a certain specific iRNA was lethal when injected into mice due to interfering with the cells ability to use its own micro RNA.

It’s important to remember that there are many different types of iRNA. While some may be harmful, many are already proving helpful. As the sorting progresses we can hopefully look forward to an era of more effective and targeted treatment.

References

- http://www.opko.com/tech.asp

- http://money.cnn.com/2007/10/09/news/companies/rna/index.htm

- http://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=484202

- http://www.bioedonline.org/news/news.cfm?art=2545

Images:

- http://upload.wikimedia.org/wikipedia/en/f/f2/SiRNA_structure_2.jpg