The University of Southampton

Author: Holly Bell

Chinese Darwin or Frankenstein? He Jiankui and the Boundaries of the Genome

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Imagine curing genetic diseases before birth. What if we could eliminate hereditary conditions, eradicate cancer or even design the perfect baby? CRISPR-Cas9, a revolutionary gene-editing tool, promises to alter DNA, with unprecedented precision. However, its immense potential raises complex ethical dilemmas.

What is CRISPR-Cas9?

CRISPR-Cas9 is the most precise and efficient gene-editing technology available. Originally part of microbial immune systems, it has been adapted for genetic manipulation. The DNA is cut at specific locations, allowing genes to be added or replaced. Unlike previous techniques, CRISPR is faster, cheaper and more accurate with applications in disease treatment, immunity enhancement and even human enhancement. However, clinical applications remain in early stages, focused on animal models and isolated human cells.

As some with a family history of genetic conditions, I find hope in CRISPR’s early success in treating diseases like Sickle Cell Anaemia. Somatic gene editing, which treats disease in individuals, holds great promise. However, germline editing remains illegal due to ethical concerns, making the dream of eradicating genetic diseases from family lines a distant vision [1].

The self-proclaimed Chinese Darwin

A medical breakthrough or reckless misuse of science? The scientist behind the first gene-edited babies presents his case.

In 2018, Chinese scientist He Jiankui made headlines using CRISPR-Cas9 to genetically modify twin embryos, Lulu and Nana, claiming to make them HIV-resistant. His experiment targeted the CCR5 gene, which also plays a role in immunity against West Nile virus and severe flu. Reports suggest the gene editing was incomplete in one twin, raising concerns of long-term health risks.

He’s work was neither curative nor medically necessary – IVF procedures had already prevented the risk of HIV transmission. Some scientists speculated disabling CCR5 could enhance cognitive intelligence, shifting the experiment from therapeutic to human enhancement. Lacking transparency and ethical approval, in 2019, He was sentenced to 3 years in prison [2].

Since his release, he has resumed research, calling himself the ‘Chinese Darwin‘; whilst critics label him ‘Frankenstein’. Unapologetic, he continues advocating for gene editing in Alzheimer’s and cancer research. His presence on social media fuels debate: is he a visionary or an unchecked egotist?

He Jiankui continues to advocate for gene editing despite global controversy. Here is what he has to say

The ethical debate

Scientific progress comes with risk. Critics warn of unknown long-term effects, unintended consequences and regulatory challenges. Most diseases are multigenic, but CRISPR-Cas9 targets single genes, limiting its effectiveness. Ethical concerns revolve around the potential of human enhancement, inequality and whether parents can truly consent to risks. Even He Jiankui admitted designer babies would be difficult to control.

Despite concerns, I support scientific progress. Why allow suffering if we have technology to prevent it? Eliminating genetic diseases would ease demand on healthcare and benefit society. Regulation, not rejection, is the key – gene editing is here and we must adapt to its evolving role in medicine. Balancing innovation with ethics will determine its future.

Looking ahead

Somatic gene editing is legal in many countries and holds promise for treating disease. However, germline editing remains controversial. As some nations ease restrictions, we may see a global divide in human genetics.

While I believe gene editing has a guaranteed future and remarkable benefits, I worry that without proper regulation, we will face a societal divide. One group will be enhanced, tailored for specific roles with predetermined superhuman qualities, from intelligence to athletic prowess. The other group will be us, free to make our own choices but facing a constant struggle to survive, and subject to natural selection.

Gene editing could revolutionise medicine, but how we choose to use it will determine whether it leads to progress or division.

References

[1] Ran FA, Hsu PD, Wright J, Agarwala V, Scott DA, Zhang F. Genome engineering using the CRISPR-Cas9 system. Nat Protoc [online]. 2013;8:2281-2308. doi: https://doi.org/10.1038/nprot.2013.143

[2] Raposo VL. The First Chinese Edited Babies: A Leap of Faith in Science. JBRA Assist Reprod [online]. 2019;23(3):197-199. doi: 10.5935/1518-0557.20190042

Custom medicine: beyond one-size-fits-all

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As medicine becomes increasingly tailored to individual needs, the one-size-fits-all approach is fading into the past. Thanks to advancements in stem-cell technology and tissue engineering, doctors are developing personalised treatments, designed for each patient. Among these breakthroughs, induced pluripotent stem cells (iPSCs) are pushing the boundaries of personalised medicine, offering therapies tailored to our unique genetic makeup. However, as this vision becomes a reality, critical questions emerge: how far can we take personalised medicine, and what hurdles stand in the way?

Stem cells in personalised medicine

Stem cells have a unique ability to self-renew and differentiate into various cells, making them invaluable in disease modelling and regenerative medicine. iPSCs, created by reprogramming adult cells to behave like embryonic stem cells (ESCs), offer a powerful tool in personalised medicine. As they are crafted from a patient’s own cells, iPSCs enable highly individualised therapies:

  • Disease modelling: iPSCs allow researches to create patient-specific cell models to study diseases and test targeted treatments
  • Personalised drug testing: scientists can predict how a patient will respond to specific drugs, minimising trial-and-error in prescribing medications
  • Regenerative medicine: iPSCs can generate patient-specific tissues for transplants, reducing immune rejection and improving long-term success
  • Gene editing: iPSCs can be genetically modified (e.g. CRISPR) to correct mutations, offering potential cures for genetic diseases

Advantages of iPSCs over past technologies:

iPSCs over embryonic stem cells

Unlike ESCs, iPSCs bypass the ethical concerns of destroying human embryos, making them a more widely accepted alternative, particularly in areas with stricter bioethical regulations. Since they are derived from the patient’s own cells, iPSCs are genetically identical enabling patient-specific disease modelling and reducing the risk of immune rejection – a major concern with ESCs.

iPSCs over animal models

iPSCs offer a more accurate representation of human disease, improving relevance and predictability of experimental outcomes, as physiological and genetic differences between species can give rise to misleading results in research. iPSCs also bypass ethical concerns in animal testing, allowing a more humane alternative approach to research and drug testing. Additionally, iPSCs rapidly proliferate in culture, allowing high-throughput screening that is difficult to achieve with animal models.

Balancing innovation and ethics

Whilst iPSCs evade the ethical dilemmas of past technologies, concerns remain:

  • Privacy: iPSCs contain sensitive genetic data that could be misused
  • Genetic manipulation: gene editing technologies may be exploited for enhancing traits rather than treating diseases
  • Inequality: the high cost of iPSC therapies could make them accessible only to the wealthy, deepening health disparities

The first success story

In 2014, Japan conducted the first clinical study using iPSCs. Masayo Takahashi led a groundbreaking trial transplanting iPSC-derived retinal pigment epithelial (RPE) cells to treat age-related macular degeneration. Whilst promising, high costs and lengthy cultivation times posed challenges. Scientists overcame this by developing allogenic iPSCs from rare donors, making iPSC therapy more accessible. In 2017, five successful allogenic RPE transplants were performed by Kobe City Medical Centre and Osaka University [1].

Looking ahead

iPSCs offer unparalleled opportunities to redefine medicine, from regenerative therapies to truly personalised treatments. This marks a promising shift away from the traditional one-size-fits-all approach, paving the way for customised healthcare tailored to each individual. While challenges remain, continued research and innovation ensure that the future of personalised medicine is bright – and this is only the beginning!


[1] https://www.amed.go.jp/en/seika/fy2018-05.html