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The Secrecy Behind the CRISPR Babies and Its Ethical Implications

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In late 2018, the world learned about the birth of Lulu and Nana, the first gene-edited twins, a revelation that remains enshrouded in secrecy. American researchers and institutions have provided vague details regarding their involvement with He Jiankui, the Chinese biophysicist responsible for altering the girls' DNA using CRISPR technology. In China, discussing the twins on platforms like WeChat can lead to censorship without any explanation or opportunity for appeal.

This lack of transparency not only obscures the fate of the twins but also conceals vital scientific information. Since late last year, He’s manuscripts detailing the creation of the twins have been under consideration by prestigious journals like Nature and JAMA, but neither has published his findings.

The main barrier to publication isn't solely due to ethical violations; He has been absent from public view for months. His lab was reportedly taken over by Chinese authorities, and his original team has disbanded. An American collaborator, Michael Deem, is currently being investigated by Rice University, but no conclusions have been released. Consequently, there may be no one available to clarify the data or perform necessary follow-up research, which is often required for journal review.

Despite widespread backlash against the CRISPR babies, the prospect of genetically modified humans is advancing more quickly than many anticipate. The techniques for genome editing are evolving rapidly, and some researchers are eager to apply them to human embryos to prevent diseases or enhance genetic traits. There is concern that similar experiments could occur in other nations with fewer regulations.

Today, MIT Technology Review is publishing excerpts from He’s unpublished manuscript for the first time, along with insights from medical and legal experts who argue that the research contains serious flaws. In a separate commentary, Kiran Musunuru, a gene-editing expert at the University of Pennsylvania, contends that these shortcomings highlight the necessity of making He’s work public.

This article will explore the journey of He’s manuscript and the ethical and legal dilemmas that have kept it from being shared with the public.

The Attempt to Publish in Nature

On the morning of November 26, 2018, He Jiankui awoke to the dawn of a media storm. News of his clandestine project to create the first gene-edited children was about to break. The twins, altered using CRISPR to disable the CCR5 gene, were born the previous month, and He had prepared a manuscript titled "Birth of Twins After Genome Editing for HIV Resistance." While he may have envisioned accolades for his pioneering work, concerns from associates about the hastiness of his actions began to surface.

By November 19, just before a significant genome-editing summit in Hong Kong, He’s team submitted their research to Nature for review. Publication in this leading journal would validate the significance of their work, allowing time for any scientific issues to be resolved. However, there were considerable gaps in the research, including the potential for unintended genetic alterations.

Meanwhile, He provided copies of his manuscripts to the Associated Press and allowed its photographers into his laboratory to gather reactions from prominent researchers. Musunuru, one of these experts, was alarmed by the data he reviewed, which indicated possible errors in the gene-editing process that were not addressed in the manuscript.

Nature, according to insiders, initially deemed the research worthy of peer review. However, He’s attempts to fulfill submission requirements ultimately derailed his publication efforts. Nature mandates that authors register any human clinical trials publicly before submission. On November 8, He retroactively registered the trial details, which had been exposed by MIT Technology Review in an article titled "Exclusive: Chinese scientists are creating CRISPR babies."

After the article's publication, He felt compelled to disclose his findings through a series of YouTube videos, explaining that the twins had been born and his motivations behind the experiment. He asserted that genetic immunity to HIV could help combat the stigma surrounding the disease in China.

In the wake of the revelation, the scientific community reacted with outrage. Prominent figures, including Feng Zhang of MIT, called for a moratorium on CRISPR-based human editing due to the lack of transparency surrounding the trials. He’s own institution, the Southern University of Science and Technology, distanced itself from the project, claiming ignorance of the research.

Consequently, on November 26, Nature’s editors decided to abandon their review, stating that the submission had become untenable after He’s institution disavowed any knowledge of the work. The journal later criticized He for disregarding ethical norms and raised questions about who could have intervened to prevent the situation.

A Chance to Go Public

Despite being turned away by Nature, He found an opportunity to share his findings with the scientific community. On November 28, he presented at the Second International Summit on Human Genome Editing in Hong Kong, displaying key results from his manuscript. As he took the stage, he acknowledged that the results had leaked unexpectedly.

Unlike other speakers, He did not provide the summit organizers with his presentation slides. However, nearly two million viewers watched online, and media crews documented the event, capturing screen images that became primary sources for scientists seeking to understand his work.

Following the summit, He returned to Shenzhen, but his team began exploring the option of posting a draft of his research on the preprint server bioRxiv, where researchers often share work before formal peer review. This platform allows for rapid dissemination of research, enabling experts to provide feedback and engage with the content.

However, bioRxiv editors informed He’s team that they might reject any submission that included unethical or illegal research practices. They needed 48 hours to make a decision, but no further communication was established.

Under Review at JAMA

He quickly pursued another publication route, submitting a manuscript to JAMA by December 3. The journal, affiliated with the largest medical association in the United States, was reportedly eager to review the manuscript.

JAMA’s editor-in-chief, Howard Bauchner, maintained an open perspective on He’s research. While some view embryo editing as ethically problematic, Bauchner believes it could eventually represent a significant medical advancement. He noted that ethical debates surrounding procedures like organ transplantation and in vitro fertilization diminished once these practices demonstrated success.

Nonetheless, JAMA took extraordinary measures to review He’s manuscript, enlisting 11 experts—significantly more than usual—to evaluate both the scientific and ethical dimensions of the research. Although He claimed to have informed trial participants and secured their consent, inconsistencies arose. For instance, it remains unclear if the twins were conceived and delivered at the same facility that approved the ethical review for the study.

JAMA’s review process has continued to be complicated by ethical concerns, leading to speculation that the manuscript may be ultimately rejected or published in an unconventional format.

A Growing Concern for Ethical Oversight

The CRISPR babies controversy has prompted journals to adopt a more rigorous ethical stance. Prior to this incident, Nature had already implemented stricter scrutiny for research involving embryo editing. In light of the scandal, there are discussions about establishing an international registry for gene-editing experiments, which would require journals to reject submissions from unregistered projects.

Experts have suggested creating a whistleblower hotline to report unauthorized gene editing attempts, which could relieve editors from making high-stakes ethical decisions alone. Magdalena Skipper, editor of Nature, expressed support for the registry initiative.

How We Obtained the Manuscript

While the manuscripts He shared with fellow researchers remained confidential, we received Word document versions of two drafts earlier this year—one detailing the twins and another discussing related embryo research. While much of the twins manuscript has been made public through media coverage and He’s presentation, the rest raises questions about whether it should be published formally or simply made available for public review.

Experts consulted on this matter expressed mixed views, but unanimously opposed publishing the research in a scholarly journal, favoring access to its contents instead. Paula Amato, an IVF specialist, emphasized the importance of maintaining ethical standards in research publication.

Ultimately, the absence of communication from He Jiankui for several months presents a significant hurdle to fully understanding the events surrounding the CRISPR babies. Rudolf Jaenisch, who was informed of He’s intentions early in the process, described the manuscript as lacking quality and unworthy of publication by reputable journals.

The ongoing debate centers on whether the complete manuscripts should be publicly accessible, enabling a thorough examination of He’s claims and the potential implications for future gene editing practices. As Musunuru notes, understanding the serious safety concerns that arose from He’s work is essential to prevent similar ethically dubious experiments in the future.

The Twins' Uncertain Future

Despite the detailed information provided in the manuscript excerpts, skeptics remain unconvinced. Many call for an independent scientific investigation to verify the existence of the edited twins and ensure their well-being.

Dana Carroll, a gene-editing scientist, emphasized the need for official confirmation from Chinese authorities regarding the girls’ existence and the validity of the CCR5 edit. The lack of transparency raises questions about the responsibilities of the Chinese state in monitoring the children and addressing public health concerns.

He’s research included plans for follow-up studies to confirm the twins' resistance to HIV, as well as monitoring their health for potential long-term effects of genome editing. However, with He’s current status unknown, it seems unlikely that these plans will come to fruition.

With reporting by Michael Standaert

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