New Location + The New and Improved Genome?

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By Young Mykhal

In the near future, your commercials may no longer be speaking about how to stop you from going bald or offering treatments for heartburn, but rather for a new type of advertisement - genetic editing. Why? It may possibly be to start preparing you for the advent of gene therapy, specifically CRISPR-Cas9, a process with the potential to cure genetic diseases. Now to say that this will actually be advertised soon is a long shot because not only is it expensive but it is far from complete. Regardless, that does not bar the fact that the advent of gene therapy is exciting, and it shows, as it is trending as possibly one of the greatest breakthroughs in medical history.  

Gene editing is not as easy as it sounds. The general definition of gene editing is that it is a process of going into your genetic code and changing how your genes are expressed with the usual intention being to make you healthier and curing you of any genetic anomalies. Take for example the genetic disorder Duchenne Muscular Dystrophy (DMD), a neuromuscular condition characterized by the wasting and weakening of muscles. What causes this is a defective form of the protein dystrophin, which normally maintains the shape and integrity of muscle cells. In DMD, this protein is not functioning properly, resulting in muscle wasting. It is a disease affecting about 1 in every 5,000 newborns, with males being the most common cases. Here is where gene therapy could come in. With the help the gene editing tool CRISPR-Cas9, diseases such as DMD could be cured. CRISPR-Cas9 stands for, get ready, “clustered regularly interspaced palindromic repeats” and Cas9 is the associated protein that cleaves DNA. In short, this mechanism is able to go to a specific sequence in your genetic code and excise DNA, potentially curing people of certain gene diseases. Why is it taking so long though?

Gene editing is a tedious process and it is far from perfect. One wrong cut in your gene and you can end up with some pretty bad results. Sometimes even with the right cut your DNA could end up repairing incorrectly, causing even more problems. Scientists now are trying to perfect this technology to increase its efficacy. Although it is not perfect, it is not to say that it has not had its fair share of successes, with one positive result being the curing of a condition called Epidermolysis Bullosa. A team of medical scientists in Italy’s Ruhr-University Bochum have managed to treat a boy suffering from this skin eating genetic disorder with help from other techniques. In short, these scientists were able to change the genes of skin stem cells (cells that can form new cells) to allow the production of new skin cells rather than the broken cells created by the condition.

Curing disease is always a great idea to look forward to, allowing us to shift further from the genetic disease paradigm medical scientists face. However, some have thought of other ways to utilize this technology that may sound unconventional and debatable to others. Human germline editing has been proposed to give children physical characteristics we desire before they are born. Is this George Orwell's Brave New World coming to life? Possibly, but not quite. Now before we start creating supermen it is important to remember that germline editing still is not invulnerable to the imperfections of CRISPR-Cas9. However, trying to regulate the editing of human germline cells may be one of the most prevalent debates when gene editing becomes a common reality.

Everyday something new is being discovered by CRISPR-Cas9, increasing its application potential. It is too early to start trying to think about how you want to change your genetic code. However, you can still perform actions to live a healthier life, like getting screened for any genetic diseases you may have or will possibly develop in the near future. Being prepared is always a great option because in this day and age, you are now empowered with knowledge of your family history and other useful information. In time CRISPR-Cas9 will hopefully outperform its other gene editing ancestors Zelda and Talens, allowing us to triumph over the many prevalent medical and environmental issues we have today.

References:

Bosley, K. S., Botchan, M., Bredenoord, A. L., Carroll, D., Charo, R. A., Charpentier, E., ...Zhou, Q. (2015). CRISPR germline engineering--the community speaks: Nature Biotechnology asks selected members of the international community to comment on the ethical issues raised by the prospect of CRISPR-Cas9 engineering of the human germline. Nature Biotechnology, 33(5), 478+. Retrieved from http://link.galegroup.com/apps/doc/A415562329/AONE?u=nysl_ca_dmvacces&sid=AONE&xid=5b843c00

Gladstone Institutes. (2018, January 18). Researchers create first stem cells using CRISPR genome activation: Activating a single gene is sufficient to change skin cells into stem cells. ScienceDaily. Retrieved January 20, 2018 from www.sciencedaily.com/releases/2018/01/180118162449.htm

Ruhr-University Bochum. (2017, November 8). Boy is given new skin thanks to gene therapy. ScienceDaily. Retrieved January 20, 2018 from www.sciencedaily.com/releases/2017/11/171108151607.htm

Nature Communications 8, Article number: 14454 (2017 doi:10.1038/ncomms14454. Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy. Retrieved January 19, 2018 from https://www.nature.com

Cherry Lam