Introduction
When does a promising technological “fix” go too far? For the field of gene editing, that question is more urgent than ever as both the promises and perils of the new technology have come to the forefront of scientific and ethical debate. While gene editing holds exciting potential to cure disease, many argue we have rushed headlong into applying it without sufficient discussion or oversight, risking unforeseen consequences that could undermine both science and humanity. As Krystal Tsosie, a geneticist-bioethicist warns that we must ensure the cure does not prove worse than the disease.
What does gene editing aim to do, and how does it plan to fix our genetic flaws? In short, it aims to precisely rewrite DNA sequences to correct mutations that cause genetic disorders or potentially enhance our genes. The technology that has accelerated this possibility is called CRISPR-Cas9, which allows researchers to precisely “edit” genes by removing, adding or altering sections of the genome. Since its discovery in 2012, CRISPR has developed at a dizzying pace due to its simplicity, accuracy and potential low cost compared to earlier gene editing tools.
The gene modification CISPR (Source: Boston Children’s Hospital)
Promises
Early successes indeed showed gene editing’s promise. Trials corrected genetic mutations in animals that cause diseases like cystic fibrosis. Researchers hoped this could lead to curing previously incurable conditions in humans by fixing DNA errors at their source. Some visionaries talk of using gene editing to make humanity more resistant to pandemics and other diseases that have plagued our species. By fixing our inherent genetic weaknesses, it seemed we may have found a way to overcome nature and diseases through technology.
Gene editing appeared poised to revolutionize medicine by allowing precise, targeted fixes for genetic disorders. From inherited conditions like sickle cell disease to more common ailments such as certain cancers caused by mutations, a wide array of previously intractable health issues seemed within gene editing’s reach. There was a lot of prediction of a future with far fewer people suffering from genetic flaws and debilitating illnesses that run in families. If proven safe through careful testing, CRISPR could potentially help cure many of the rare diseases caused by a single gene mutation including cancer, Alzheimer’s disease, HIV, autism, muscular dystrophy, and inherited eye disorders. The potential societal and economic benefits of gene drives to control agricultural pests or mosquito-borne diseases also excited supporters.
However, the full promise of gene editing fix remains uncertain until more research can explore applications and long term effects. While early animal tests proved encouraging, human trials will take considerably longer and face complex ethical hurdles. Any premature scaling from animals to people could endanger safety, as the “CRISPR babies” fiasco demonstrated. Continued open discussion will thus be important to guide responsible development of this powerful new biotechnology.
Concerns
However, as with any new technology applied in biology, unintended consequences can emerge that were difficult to predict. With CRISPR there are concerns about off-target mutations where the editing goes awry and inadvertently changes other parts of the DNA. Such issues could have unforeseen health impacts that may not emerge for generations. Since CRISPR edits in the germline — reproductive cells and early embryos — this raises ethical claims about permanently altering the human gene pool in ways we may not understand for decades or centuries to come.
Some argue this path could open a door to eugenics and “genetic enhancement” that most feel should not be pursued. A lot of scientists fear gene editing could worse social inequities by allowing the wealthy earlier access to genetic fixes, or even drive desires to alter children for non-medical reasons like traits or abilities. Questions also remain about informed consent in making heritable changes to future generations who cannot consent themselves. These concerns came to a head in late 2018 when Chinese scientist He Jiankui shocked the world by claiming he used CRISPR to genetically modify twin baby girls, altering their DNA to purportedly make them resistant to HIV. It showed how premature and cavalier application of gene editing for human reproduction could bypass regulations and safety testing.
The Debate Grows
As incidents like the “CRISPR babies” case in China took place, discussions around gene editing intensified across the scientific community and broader public. Many researchers felt that more oversight was urgently needed to ensure safety standards were followed. While the promise of curing disease drove some to push boundaries, others warned against premature applications without adequate review and consent procedures in place.
Genetic editing (Source: Gao)
At a conference in Hong Kong in 2019, several scientists debated whether a moratorium should be imposed on any clinical uses of gene editing for heritable DNA changes until governance caught up. Opinions varied, as some felt this could hamper research, while proponents of a pause argued lives were at stake if experiments moved forward without oversight. This debate crossed borders as different countries adopted different policies. The UK allowed gene editing of human embryos for research, but not for clinical purposes. France banned gene editing of the human genome that could be inherited. China and India had weak regulations that enabled some scientists to conduct risky experiments. As discussions intensified on this topic, it became clear there were no simple answers. Both progress and safety had to be balanced according to many involved. But doing nothing risked lives through premature testing, while over-regulation threatened to slow medical advances that could save many from disease. It seemed the only consensus was that more discussion with all stakeholders was essential to find responsible solutions.
A Fix That Backfired?
The saga of He Jiankui’s experiment in China served to reinforce many critics’ warnings that premature applications of gene editing risk serious damage, both to science and the human subjects involved. By bypassing proper oversight and safety testing, it undermined public trust in whether this technology could be developed responsibly. Some argue we have rushed headlong into heritable human experiments without sufficient discussion of ethical and long-term issues, prioritizing headlines over careful progress.
The case highlighted how without international consensus on governance, rogue scientists may conduct dangerous studies for their own fame and fortunes that endanger lives. It also showed gene editing of human embryos remains unpredictable, as the intended genetic modifications and long-term health effects on any resulting children cannot be known. While He claimed to use CRISPR to make the twin girls resistant to HIV, effects on their overall health and fertility are impossible to foresee. Such uncertainties renew concerns about prematurely tinkering with the human germline.
As many experts warned, premature or dangerous testing could severely damage public trust in science and medicine at a time when it was needed most. Some felt gene editing may have already backfired and undermined its own promise through rash applications pursued before risks were adequately discussed and addressed. Only time would tell if oversight could be implemented before further controversies did lasting damage.
Conclusion
While gene editing was developed with the promise of using technology to “fix” inherent human genetic flaws and cure disease, research shows both the immense potential and serious risks of this approach remain unclear. As the field rapidly progresses, discussions have shown the need for international cooperation to ensure its applications proceed safely, ethically and with appropriate oversight that maintains public trust. How to balance innovation, ethics and governance will likely continue to stir complex discussions. If developed responsibly and inclusively with input from varied stakeholders, gene editing may yet fulfill its goals. However, more care is clearly needed to ensure the promised “cure” does not prove worse than the disease.