CRISPR Dreams: The Potential for Gene Editing

Bonny Lemma. Originally published in the HIR Winter 2019 Issue.

Jennifer Lopez has one more industry to add to her illustrious résumé: molecular biology. In 2016, she was asked to be the executive producer of a new futuristic bio-crime drama for NBC called C.R.I.S.P.R. While that project is a work of science fiction, the CRISPR technology that it is based on is very real.

CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is not just a gene editing technique, but also a phenomenon that carries significant implications for the future of biotechnology. Therefore, the interactions between the countless players in this field and the objectives driving them are crucial to understanding of CRISPR and the promise it holds.

Dr. Richard Losick, professor of biology at Harvard University, once said that “Day science, how one presents their discoveries, has the linearity afforded by reflection, and night science, the reality of the discovery process, is a messy, progressive, questioning construction and destruction of hypotheses.” His reflection encapsulates the winding road of CRISPR experimentation. In 2002, Spanish biologist Francisco Mojica and Dutch biologist Ruud Jansen recognized distinct repeating motifs in about 50 DNA base pairs, neatly interspersed through bacterial and archaeal genomes that were non-coding but too ordered to be insignificant. 10 years later, Martin Jinek proved that a specific type of RNA had the ability to cut apart invading DNA. His discovery spurred an outpouring of research in CRISPR technology; by the end of 2015, researchers published five papers per day regarding CRISPR.

The scientific community is simultaneously a cooperative and competitive arena. In 2017, researchers at the University of California Berkeley sued their MIT counterparts over small distinctions between their respective gene editing patent. Despite these occasional squabbles, the field is better characterized as one of radical cooperation. Today, genome engineering expert subcontractors are readily available to help labs around the world outsource their CRISPR-Cas9 analysis to work more quickly and efficiently. In the rapidly growing and intensely experimental field of molecular biology, information and sample sharing has been a boon to labs everywhere.

Researcher Feng Zhang published a paper on the gene editing function of CRISPR; rather than FedExing his vials of DNA to every lab that requested them, he uploaded his CRISPR code to Addgene, a nonprofit molecular biology sample sharing service. The company collects, manufactures, stores, and sends out DNA fragments to academic labs and other nonprofits, helping more and more scientists every year. The organization removes the hassle and inefficiency of one-on-one lab requests for samples, a process that often took a long time and was often inaccurate. Since its inception, the lab has shared more than 42,000 CRISPR components with thousands of scientists across dozens of countries.

Still, all legislative climates are not created equal; good policy is an important factor in the growth of this frontier of biomolecular science. The US National Academies of Science has hosted domestic and international meetings to discuss the positive and negative implications of gene editing. In 2016 and 2017, the practice of gene editing was listed in the Worldwide Threat Assessment reports submitted to Congress by the Office of the Director of National Intelligence. However, the rapidly changing nature of CRISPR research makes it difficult for governments and industry leaders to consistently update their regulations. This failure has led to dangerous misunderstandings of the technology that fail to anticipate the next discovery’s implications.

Debates over CRISPR are ongoing, but the many possible applications have grabbed the attention of research doctors, agriculturalists, and epidemiologists alike. Researchers at the University of Washington used CRISPR-based gene replacement therapy to treat the effects of Duchenne muscular dystrophy in adult mice. They were able to restore muscle strength in the mice after just four months of treatment. Agricultural biotechnologists demonstrated that CRISPR could be used to modify genes associated with certain crops’ susceptibility to bacterial infection. Gene editing even has the capacity to drive infectious species to extinction, including the most lethal animal on Earth, the malaria-causing Anopheles gambiae mosquito.

Unfortunately, public discourse has overlooked the benefits of CRISPR technology and centered instead on the possibility for more controversial applications. A Google search on the topic of CRISPR technology yields thousands of articles with lurid titles like “11 Crazy Gene-Hacking Things We Can Do with CRISPR,” “Mail-Order CRISPR Kits Allow Absolutely Anyone to Hack DNA,” and “CRISPR in 2018: Coming to a Human Near You.” While the scientific community continues to make progress and governments around the globe continue to grapple with regulation, pop science has sensationalized this crucial technological breakthrough. Unsurprisingly, the public has railed against the use of human embryos in clinical trials and the possibility of “designer babies.” Public perception of CRISPR is shaped a great deal by this sensationalism. A misinformed populace is inimical to serious conversations about the technology and its limitations.

Those conversations cannot happen quickly enough. If CRISPR research can refocus, the technology is poised to alleviate the major resource and medical obstacles facing the world today. Organizations have not fully realized CRISPR’s potential. For example, the agricultural technology industry has incorporated CRISPR technology into its “superfood” laboratories, without investigating the types of foods that would support the most people. Such experiments should be auxiliary applications of this technology, which has far more potential to help alleviate malnutrition and economic depression. In the same vein, those who fret about designer babies overlook the massive benefits that could come from further research into more efficient treatments for communicable diseases like HIV, malaria, tuberculosis, cholera, and even Ebola.

Whatever priority issues arise on the research side of CRISPR technology, funding will be crucial. The US government announced a US$190 million budget for the National Institutes of Health (NIH) to award research grants over the next six years. This could have profound implications for the future of CRISPR research. The NIH funds will go to the Somatic Cell Genome Editing program. According to Francis Collins, the director of the NIH, this will “dramatically accelerate the translation of these technologies to the clinic for treatment of as many genetic diseases as possible.” Though technically reserved for genetic diseases, the grant will allow researchers to develop better delivery mechanisms, which could be critical for fighting communicable diseases.

Similarly, the Department of Defense is putting US$65 million into research to improve CRISPR and protect against its potential weaponization. Like the NIH money, the grants funded by the Department of Defense will address problems relating to delivery systems and treatment generalization, specifically targeting viral diseases like Ebola and Zika. It should be noted that this grant and its research may have more strings attached—strings that may suppress the radical information sharing that nurtured the early growth of this industry. Still, the mere acknowledgment of CRISPR as a viable technology is an important step forward.

When resources and priorities clash, the scientific big picture can get blurry. CRISPR’s relative novelty has afforded it a comfortable shelter from this conflict so far. But as time goes on, the cooperative spirit may start to disintegrate because of researchers’ and politicians’ competing objectives. Further, there remains the possibility that this brilliant but flashy new technique will overshadow the most pressing challenges that the international community faces today. And yet, the impatient public may influence the crucial discussions that have yet to take place. In spite of all of these imposing possibilities, the beauty of this scientific moment cannot be overstated. CRISPR has certainly captured the hopes and curiosities of the world. Even if the fruits of its promise lay far in the future, the world at least has one more procedural crime drama on the way. The next new CRISPR development might come out in the papers or on primetime. The world will just have to stay tuned to find out.