Editorial: Gene editing is an opportunity for chemistry’s good governance

Researcher He Jiankui made headlines in 2018 when he announced that he had created the world’s first babies with genetically edited DNA using the CRISPR-Cas9 technology. His revelation shocked the scientific community. Many believed Jiankui’s work was an ethical violation.

The Nobel Prize awards provide an opportunity to contemplate on this ethical dilemma as we reflect on winners past and present. In 2020, the CRISPR-Cas9 work of duo Jennifer Doudna and Emmanuelle Charpentier won them the prize in chemistry. This year, a winning duo in the physiology or medicine category for work on microRNA brings advances in genetics research back into focus. In 2019, Jiankui was found guilty of “illegal medical practices” and sentenced to 3 years in prison. In July this year, China’s Ministry of Science and Technology prohibited all clinical research involving germ-line editing. While the explicit prohibition is welcome, guidelines alone may not stop unethical research. The science community must also step up to make such behavior unacceptable.

Getting the regulation right on gene editing’s dual-use potential requires careful but urgent thought.

The development of a DNA-editing tool such as CRISPR-Cas9 has been an incredible technological achievement, with myriad possibilities for treating diseases. It could treat those with debilitating genetic disorders caused by single gene mutations, such as cystic fibrosis, hemophilia, and sickle cell disease. In December 2023, the US Food and Drug Administration approved the first CRISPR-Cas9-based cell therapy gene therapy for treating sickle cell disease. The treatment modifies a person’s blood stem cells, which are then transplanted back into the person’s bone marrow.

While gene editing technologies have the potential to treat several human diseases, its unethical and unapproved use could have far-reaching and perhaps immutable consequences for society overall.

The blood stem cells edited to treat sickle cell disease are somatic cells, and subsequent generations don’t inherit the edits made in them. But Jiankui’s work focused on permanent germ-line cells, which future generations carry forward. Editing germ-line cells has several risks; for example if anything goes wrong, the changes are permanent and could propagate to future generations. Meanwhile, errant actors in the field could use the technology to enhance desired traits, opening doors to the horrors of eugenics.

In March 2023, the Organising Committee of the Third International Summit on Human Genome Editing published a statement concluding that “heritable human genome editing remains unacceptable at this time.” The group noted that we lack a governance framework and ethical principles and that the technology hasn’t met safety and efficacy standards.

Educating the public and involving people in conversations around such technologies’ social, ethical, and legal implications is crucial for facilitating public trust in science. Although some studies investigated people’s perceptions of these new technologies, there aren’t enough data to understand how the public perceives different forms of gene editing and thinks it could affect lives.

Countries must urgently deliberate on the topic and create a legal framework that results in better transparency and regulatory oversight. Doudna told C&EN in 2018 that introducing new technology is like opening Pandora’s box: “You can’t close the box again,” she said. “It just requires that the folks involved in the original science actively engage in a discussion of how to regulate it, how to self-regulate it, and how to appropriately steward it in the future.”

As DNA-editing technologies such as CRISPR-Cas9 spread to newer applications, Doudna’s word of caution hits home. And the chemistry community would do well to enact measures to follow her advice on regulation.

This editorial is the result of collective deliberation in C&EN. For this week’s editorial, the lead contributor is Aayushi Pratap.

Views expressed on this page are not necessarily those of ACS.