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El Bat Cate School

An Introduction to CRISPR Gene-Editing

Sebastian Sutch ‘24



For the vast majority of human history, individuals born with hereditary diseases and genetic mutations have been regarded with a sense of fear and uncertainty, consigned to the margins of society. Before the scientific revolution of the 16th century, wherein the field of biology was pioneered, and the study of human anatomy was conducted with proper, precise methodology and technology for the first time, illnesses were seen as divine punishments—a physical expression of God’s might, anger, and discontent (Roy Wenzel, How Diseases Spread: Ways People Have Tried to Explain Pandemics Through History, History). As a result, individuals who were impacted by then-inexplicable afflictions were forced to confront genuine social stigma, as it was thought that mere interactions with these ailing individuals had a contaminating, harmful effect. Thus, the plight of the sick was twofold; in addition to having to simply endure their respective maladies, these individuals were frequently subjected to unabashed discrimination, and, as such, lacked a pathway to live normally. Fortunately, the advancement of modern medicine has disproved and debunked the myriad false convictions that ancients held regarding the nature of illnesses. There now exists a scientifically reinforced explanation for the cause of Alzheimer's, Parkinson’s, and virtually every other major hereditary disease. Yet, despite this remarkable intellectual progression, the treatment which the individuals who actually suffer from these illnesses haven't improved at the same rapid rate. However, CRISPR gene editing offers a potential paradigm shift—one that may render hereditary diseases and genetic mutations a relic of the past, or one that may change the very definition of what it means to be human.


Discovered in 1987, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeat) is a family of specially-stretched DNA sequences—the molecule which contains the genetic code of each living organism—that behave like a pair of “molecular scissors, capable of cutting strands of foreign, unwanted DNA” that is found in nearly every living organism (Aparna Vidyasagar, What is CRISPR?, LiveScience). This enzyme can be effectively leveraged by scientists to alter DNA and gene sequences; once this occurs, the cell’s natural responses are activated, and the desired changes are rendered permanent.


Considering CRISPR’s unprecedented ability to modify the cell’s biological makeup, though, its impact upon global society has, thus far, been fairly minimal. In fairness, the enzyme in CRISPR was only first ‘hijacked’ by scientists in 2012, so technology’s limited social infiltration can largely be attributed to its youth. In regards to its current applications in humans, researchers at the University of Pennsylvania have begun trials to observe CRISPR’s usage in the treatment of multiple myeloma, a cancer of the blood and bone marrow, but results have been fairly inconsistent (Michael W. Richardson, What is CRISPR Currently Being Used For?, BrainFacts). The number of such trials taking place across research facilities around the world is growing daily; from cancers like leukemia and Hodgkin's lymphoma to autoimmune diseases like sickle cell disease, the technology has shown genuine promise in some studies. Interestingly, CRISPR’s usefulness in comparatively less important, non-human contexts is significantly greater; it has been successfully used to increase cocoa trees’ protection and resistance against otherwise-deadly diseases. A gene-edited tomato plant has also been created using CRISPR; aside from being unnaturally compact, it also yields fruit early (Chuck Gill, Cocoa CRISPR: Gene editing shows promise for improving the 'chocolate tree', Penn State). Generally speaking, though, CRISPR’s massive potential hasn’t been realized or unlocked. While the technology’s capacity to eradicate disease and mutation is certainly attractive, CRISPR’s use in modifying the biological makeup of humans—as opposed to simply in crops or livestock—is incredibly dangerous, and may have ramifications that will be felt forever.


In November 2018, He Jiankui, a Chinese biophysicist, shocked the world. Speaking at a gene-editing conference in Hong Kong, he revealed that he had helped to produce history’s first genetically modified human. Prior to their birth, Jiankui used CRISPR to modify a pair of identical twins’ embryos such that they became resistant to HIV. While Jiankui claimed to be motivated by wholly altruistic intentions, his announcement sparked outrage from across the globe. Numerous scientific societies from Jiankui’s native China were deliberate in condemning the dangerous, reckless nature of his study, including the Chinese Academy of Sciences, who stated that Jiankui “[violated] internationally accepted ethical principles regulating human experimentation and human rights law” (David Cyranoski, CRISPR-baby scientist fails to satisfy critics, Nature). Within months of Jiankui’s announcement, he was arrested by authorities, found guilty of illegal medical practices, handed a hefty $3 million Yuan fine and a 3-year jail sentence. While justice was supposedly served, Jiankui’s experiment raises numerous questions, some more immediate than others.


1) Regarding Safety & Legal Regulations:

Even following Jiankui’s rashly conducted experiment, no international body has created a set of unambiguous guidelines that detail the manner in which CRISPR should be used. Language in legal documents remains vague and unclear, which means that individuals like He Jiankui continue to exploit glaring loopholes to perform potentially hazardous procedures. While 40 countries have released statements that either discourage or ban scientists from engaging in gene-editing-related research, proper oversight is extremely minimal. Even following the Second International Summit on Human Genome Editing, wherein over 500 scientists from across the world convened to propose a standard for CRISPR regulation, this issue hasn’t been resolved. The summit deemed its usage in human applications to be “irresponsible”, but it arrived at no regulatory conclusions. Rather, it merely concluded that given the “fluid,...evolving” nature of ethical considerations, regulating CRISPR would be impossible until the “[establishment of] a body that would define a global...code of conduct, which could in turn support oversight, approval, and consent” (The National Academies Press, Second International Summit on Human Genome Editing: Continuing the Global Discussion Proceedings of a Workshop—in Brief, The National Academies Press). As mentioned above, CRISPR is a largely untested, volatile technology. Promising, yes, but unproven nevertheless. In fact, a 2019 study aimed at fixing defective DNA in human embryos showed that in half the instances in which CRISPR was used, the editing caused unwanted, dangerous changes; in some instances, entire chromosomes were inadvertently removed as a result of a botched procedure. The international community’s continued passive approach to CRISPR regulation may soon have serious ramifications; a human toll due to gross scientific and governmental negligence may soon arise. Thus, it’s imperative that a legislative body acts decisively, and establishes a standard by which researchers should be expected to abide by. Of course, these regulations shouldn’t render CRISPR completely obsolete; there will certainly come an age when the technology will better society, but for now, considering its unreliability, its applications should be heavily restricted.


2) Regarding Justice, Equity, and the Contamination of the Human Gene Pool:

As CRISPR’s precision and prevalence continue to increase, there is a growing concern amongst the scientific community that given the cost-prohibitive nature of genetic engineering, only wealthy individuals will be afforded the opportunity to gain absolute immunity against all diseases and mutations. In so doing, existing disparities in healthcare—wherein the cost of treatment generally corresponds to its quality—could be compounded, thereby leaving underprivileged individuals at a substantial disadvantage. Taken to a hypothetical extreme, where CRISPR might be used to correct all genetic traits, like height or athleticism, a dystopian class system in which individuals were entirely defined by the extent and quality of their modified genetics could be created. If genetic traits could be entirely determined by CRISPR prior to the birth of a child, would they possess any individuality whatsoever? In addition, if CRISPR is used to make genetic edits to an embryo, sperm cell, or egg, the corrections are permanent. Essentially, this means that all future generations will inherit the characteristics and changes that one individual decided to acquire. This ripple effect means that eventually, the entire human race could bear some marking or indication of genetic editing. Ultimately, CRISPR poses a question regarding the very essence of what it means to be human; if all traits could be artificially implanted, what characteristics would individuals be defined by?


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