In the aftermath of decoding human DNA, thoughts turned to editing genes as the primary means of changing disease outcomes. But as it turns out, cancelling gene expression may be a less risky and less controversial treatment in many cases. Think of it as the difference between using surgery to remove or modify genes versus using noise cancelling genetic headphones where gene expressions are suppressed so they no longer bother you.
That is, of course, a gross over-simplification in comparing the two approaches in treating diseases at the genetic level. But that metaphor is helpful in quickly wrapping one’s mind around two extraordinarily complex ideas.
However, details matter in truly understanding anything, so start with a look at how genes express themselves and shape our health realities. Here is a short and easy to follow video to explain what gene expression is:
Now, getting marching orders to cells in this way sounds like everything is perfectly regulated, and most of the time it is. After all, cells contain the same DNA so controlling which genes work in which cells is vitally important to the overall organization of the organism, or you know, functioning creatures such as humans! Good health means all cells, or at least the vast majority of them, are functioning correctly.
Gene regulation determines which genes are turned on and which are turned off, in any given cell. This process isn’t as simple as toggling a light switch. If you want examples in laymen language on how gene regulation works, watch this short, animated video:
Increasingly, research is pointing to several options in treatments at the cellular and genetic levels. Two or more are often combined to leverage the benefits of each.
Immuneering is one company that uses bioinformatics to develop new medicines as well as innovations for existing medicines based on emerging genetic techniques. Co-founder and CEO Ben Zeskind describes many of the newer drugs to be like noise-canceling headphones.
The company “calls it ‘disease-canceling technology,’ which analyzes the gene expression data of diseases and uses computational models to identify small-molecule compounds likely to bind to disease pathways and silence them,” according to an article in SciTech Daily.
“Our most advanced candidates go after the RAS-RAF-MEK [protein] pathway,” Zeskind explains in that same article. “This is a pathway that’s activated in about half of all human cancers. This pathway is incredibly important in a number of the most serious cancers: pancreatic, colorectal, melanoma, lung cancer — a lot of the cancers that have proven tougher to go after. We believe this is one of the largest unsolved problems in human cancer.”
As research progresses, companies are discovering more ways to defeat inherited and acquired diseases in varying genetic and cellular techniques.
“As long as we have a good gene-expression signature from human patient data for a particular disease, we’ll find targets and biological insights that let us go after them in new ways,” Zeskind says in the SciTech Daily article. “It’s a systematic, quantitative, efficient way to get those biological insights compared to a more traditional process, which involves a lot of trial and error.”
In short, discoveries are happening quickly and there’s lot of reasons for patients with varying diseases to feel new hope for a cure. As the research continues, take a moment to appreciate the advances that have already been made. This video is a quick explanation of the difference between gene therapy, cell therapy, and gene editing:
Here’s to good health for all in the foreseeable future as bioinformatics continues to open the door wider for new medical possibilities!