The Herman Trend Alert
February 17, 2016
Genetic Modification to Save Lives
In the United States, we have long outlawed genetic modification for our belief is that it allows us to "play God." Yet the development of efficient and reliable ways to make accurate changes to the human genome has remained a goal for research for years.
From bad comes good
Not long ago, a new instrument, based on special bacteria, generated a lot of buzz. Using a particular part of an avowed enemy of humankind Streptococcus pyogenes, CRISPR*-associated protein-9 nuclease (Cas9), researchers have been remarkably successful (1). Streptococcus pyogenes is the nasty class of bacteria that causes what we call "strep throat," as well as scarlet and rheumatic fevers and even tonsillitis.
How CRISPR/Cas9 really works
The functions of CRISPR* and CRISPR-associated (Cas) genes are vital for select bacteria (and the bacteria from which they are derived) in something called "adaptive immunity," enabling the organisms to respond to and eliminate invading genetic material---very valuable. Basically, CRISPR is the simply the most versatile gene-editing tool on the planet.
The real breakthrough
Three years ago, Professor Stanley Qi now at Stanford University, found a way to stop CRISPR from editing any genes. What resulted is that "the dead enzyme can now act as a platform for other molecules, including activator molecules that switch genes on, repressors that turn them off, or glowing substances that reveal their locations." And according to The Atlantic Magazine, with the addition of the right "guide molecules," scientists can now direct these payloads to any gene they like. This process results in a "precise and versatile delivery system, which can control any gene you want." Now, it is not merely "an editor," but rather a platform that may be used in a myriad of exciting ways. Some of these exciting uses might be to use gene therapy to reactivate heart muscles after a heart attack, or to stop genes that promote the growth of cancers. Or even to stop viruses dead in their tracks! Once stopped, the immune system can clear it. Then they can turn the genes back on and the person is back to normal.
The downside
Since Cas9 comes from bacteria, having it in the body for a long time exposes the person to having a horrible immune response---not a risk worth taking in many cases. But for serious cancers and auto-immune diseases, using this system is very logical.
The future of CRISPR/Cas9
Researchers have made remarkable rapid progress in developing Cas9 into a set of tools for cell and molecular biology. This speed probably due to its ease-of-use, high efficiency, and versatility. Of similar systems now available for precise genome engineering, the CRISPR/Cas system is by far the most user friendly. It is now also clear that Cas9's potential reaches beyond DNA dissection, and its usefulness will likely only be limited by our imagination.
* CRISPR=Clustered Regularly Interspaced Short Palindromic Repeats
References
1. Cong L., et al. (2013) Science, 339, 819-823.
Special thanks to The Mensa Bulletin for raising our consciousness to this important breakthrough.
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