He Jiankui, an independent Chinese researcher, recently triggered global controversy and confusion over claims that his experiments produced the first genetically altered babies using gene editing technology. The scientist claims to have used CRISPR/Cas9 technology to alter the DNA (deoxyribonucleic acid) of embryos before implanting them into the mother’s womb to make the twin babies resistant to HIV. The unverified claim by He—propagated through media and online videos—has stoked public fears and renewed apprehensions that babies might one day be “designed”. It raises an urgent need for sound governance and greater public dialogue on gene editing.

Shruti Sharma
Shruti Sharma is a research analyst with the Technology and International Affairs Program at the Carnegie Endowment for International Peace. She works primarily on the safety, security, and ethical implications of emerging biotechnologies.
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While it is illegal to deliberately alter the genes of human embryos in India, in the US, and many other countries, the legal position on gene editing in China is less clear. However, within the scientific community in China, He’s claims were openly chided. Some Chinese scientists made clear that He’s claims were “a huge blow to the global reputation and development of Chinese science”.

The broader scientific community condemned the lack of transparency in the development, review, and conduct of clinical procedures for He’s experiment. Two notable failings of this experiment were the inability to obtain consent from the participants of the trial, and the highly questionable ethical standards implemented to protect the welfare and rights of the research subjects.

Nikhil Sahai
Nikhil Sahai is research intern at Carnegie India.

Gene editing experiments may prove to be a blessing for parents carrying disease-causing mutations to have their own children, yet these interventions raise crucial safety and efficiency concerns leading to what scientists and doctors call off-target mutations and mosaicism. In the former situation, CRISPR could miss the target gene and attach itself to another similar sequence, thereby creating properties far different from those that were intended. In the latter scenario, whereas CRISPR connects to its target gene, it could well alter the DNA sequence of only some, and not all, of the necessary cells. This may lead to genetically distinct cells, whereby some cells may still carry the deadly mutation, rendering the treatment ineffective. Since the results of He’s experiment have not been published or peer reviewed, some experts fear that his effort might not have been screened for off-target effects and mosaicism, therefore putting the twins’ health at risk.

The uproar over He’s experiment calls into sharp contrast the incredible potential and possible pitfalls of gene editing. These technologies hold the promise of curing any human genetic disease, triggering a global sprint to translate potential therapies into clinics. Switzerland-based CRISPR Therapeutics, with labs in Massachusetts, and Boston-based Vertex Pharmaceuticals have recently launched human trials of an experimental CRISPR-Cas9 therapy for b-thalassemia, a blood disorder that decreases the production of haemoglobin, an iron-containing protein in red blood cells that carries oxygen. Moving quickly to develop gene therapies, an oncologist at China’s Sichuan University was the first to edit human cells to treat lung cancer in 2016. China is also attempting to halt disease progression in patients with oesophageal cancer by manipulating a piece of DNA in white blood cells.

But these advancements in therapeutic genome editing have been called into sharp focus following He’s experiment. Crucial questions need to be asked with regards to fragmented legal frameworks, unclear regulatory practices ambiguous policy advances and voluntary measures governing gene-editing technologies at national and international levels. Considering the rapid pace of genome editing, the existing overarching governance frameworks in India and elsewhere need urgent examination and development.

Further, the lack of informed public knowledge about genome editing (GE) and how it differs from genetic modification (GM) can fuel misconceptions about research, applications, and risks of genome editing. While GM involves permanent integration of a foreign gene into the host genome, GE only involves manipulating the endogenous gene without inserting a foreign DNA.

So, in addition to drafting regulatory frameworks, it is important to exchange decision relevant information including scientific, ethical, regulatory and political considerations of GE advancements to society. Communication should not be confined to the educated segment of the population but reach a wider section of society. Independent advisory groups and consultation committees can and should be engaged to ensure legitimacy of the public engagement effort. These independent consultants can reach out to patient advocacy groups, religious committees, groups concerned with women and gender rights, environmental activists, media agencies and government to reach the most number of relevant public and policy actors.

Feedback and solutions suggested by the public must be effectively communicated to regulatory agencies and policymakers at the central and state levels to develop a comprehensive framework to regulate genome editing technology.

In conclusion, the process of modernizing existing governance frameworks should be complemented with public engagement efforts aimed at closing knowledge deficits and building relevant scientific literacy among non-expert audiences. This collaborative effort will help ensure safe, secure, and responsible uses of biology tied to societal needs.

This article was originally published in the Livemint.