Is CRISPR really a gene-ius discovery?

Since the discovery of its gene editing potential less than a decade ago, the CRISPR-Cas9 system has been a hot topic of debate.  (RF/Flickr, Creative Commons)

Since the discovery of its gene editing potential less than a decade ago, the CRISPR-Cas9 system has been a hot topic of debate. (RF/Flickr, Creative Commons)

Since the discovery of its gene editing potential less than a decade ago, the CRISPR-Cas9 system has been a hot topic of debate. As scientists grow closer to unlocking its possibilities, ethical questions regarding the topic are only increasing. Now that human trials have officially begun in the United States, the scientific community at large must quickly come up with the answers to these questions. It is therefore of the utmost importance that increased regulations be set in place now to govern the usage of this powerful tool. 

I remember when I first learned about CRISPR-Cas9 in my AP Biology class in high school. My teacher explained that the Cas9 enzyme naturally occurring in bacteria essentially works as scissors, recognizing and cutting apart viral RNA. Our class then attended a genetic conference, where I marveled at the speaker’s illustration of CRISPR’s simple yet incredible ability to cut away and replace mutated DNA in the same way. He was moved to tears at the prospect of using this discovery to repair the single nucleotide mutation that causes sickle cell anemia or the defective gene in type I diabetes. I walked away from the event in awe, but was promptly brought back to Earth by the realization that this technology was not yet being used on human subjects in the U.S. I was still intrigued by the topic, but I assumed that it would be many years until scientists could actually add CRISPR to their disease-fighting repertoire. Little did I know that the University of Pennsylvania would treat two patients with CRISPR in April of 2019, only two years later.   

Researchers at the University of Pennsylvania have devised a new cancer cell treatment in which they modify patients’ extracted immune cells with CRISPR and then re-implant the cells. The University of Pennsylvania is not the only organization beginning CRISPR human trials; several burgeoning studies are also emerging across Europe, the United States and Canada. However, with the rapid growth of testing on human subjects, it is essential that extreme caution is exercised moving forward. 

Although it has been fully sequenced, there is still much that remains unknown about the human genome. Editing one DNA sequence with the intent of curing a disease could result in a different, unintended health issue, potentially even worse than the first. Additionally, manipulation of human embryos could pass changes along to following generations, including any unknown issues. While not currently allowed, the possibility of germline edits has not yet been ruled out, leaving the door open for genetic alterations to leave a lasting imprint on the future, good or bad.  

Another area of concern with the human trials is the level of inspection that they must be subjected to prior to initiation. The National Institutes of Health (NIH) and the Food and Drug Administration have recently modified their requirements, removing the additional review of all gene editing studies previously needed. The Recombinant DNA Advisory Committee created by the NIH to monitor all genetic experimentation will now only review gene therapy protocols that are deemed high-risk. This more relaxed policy applies to the current and future human CRISPR trials, but should not be taken as an excuse for experimenters to lower their guards. 

As the wise philosopher Ben Parker once said, “With great power comes great responsibility.” Scientists would do well to keep this phrase in mind as CRISPR research marches forward. With trials launching worldwide, we can only wait with anticipation of what this research will yield. Nevertheless, approval of CRISPR trials does not give members of the scientific community permission to sit back and observe the results, hoping for the best. Further guidelines must be implemented to regulate future CRISPR use and researchers must be held to the highest standards to ensure that they tread carefully in their experimentation on humans. 


Veronica Eskander is a campus correspondent for The Daily Campus. She can be reached via email at veronica.eskander@uconn.edu.