In 2018, He Jiankui, a Chinese researcher, made headlines by creating the world's first gene-edited babies, Lulu and Nana. He claimed to have edited their genomes to make them resistant to HIV, but his work was met with widespread criticism and ethical concerns.
Uncertain Outcome
A major concern was mosaicism, where the gene edits were not uniform across the twins' cells. This means some cells might be edited, some not, and others partially edited. Additionally, He only managed to edit one copy of the CCR5 gene in Lulu, making her either heterozygous or mosaic for the edited gene. This raises doubts about whether the twins are truly resistant to HIV.
Off-Target Edits and Unintended Consequences
Further analysis revealed He's edits were not as intended. He aimed to mimic the naturally occurring delta 32 mutation, but the twins ended up with entirely different mutations. These mutations are untested and could have unknown consequences, including cancer and heart disease. Additionally, the possibility of off-target edits raises concerns about unintended changes to other genes, which may even be passed on to future generations.
The Need for Transparency
Despite the ethical concerns and potential risks, He's work remains largely unpublished. This lack of transparency hinders the scientific community's ability to understand the full scope of his experiment and learn from it.
AI's Crucial Role
AI played a critical role in analyzing the twins' DNA and identifying issues like mosaicism and off-target edits. This information was essential in highlighting the potential risks associated with He's work.
Moving Forward
The He Jiankui case underscores the urgent need for transparency and ethical guidelines in the field of human germline editing. International committees are working to establish regulatory frameworks, but this can only be effective with full disclosure of He's research. By making his work public, the scientific community can learn from his mistakes and prevent similar incidents in the future.
Preventing Future Incidents
With individuals like Denis Rebrikov pushing the boundaries of human germline editing, transparency is vital to ensure oversight and risk assessment. Just as the disclosure of resurrected horsepox virus raised concerns, He's work serves as a cautionary tale for the scientific community. Publishing his research is crucial to prevent further unethical and potentially harmful experiments.
Conclusion
The story of Lulu and Nana raises significant ethical and scientific concerns about human germline editing. Transparency and open discussion are essential to ensure the responsible development of this powerful technology. By learning from the past and working together, we can build a future where gene editing is used for good.