It’s been described as a cut-and-paste tool for DNA and hailed as the most ground-breaking and important discovery of our time. But the incredible advancements in gene editing through CRISPR has inevitably sparked controversy in the process. If we have the means available to us, should humans “play God”?


Legislating in these ethically muddy waters is already proving a challenge. If we set the parameters for genetic editing to be limited to use in preventing disease in embryos, where do we draw the line? What constitutes as a critical intervention and when does it err into the territory of designer babies?


Once upon a time, the ability to fundamentally change the genetic sequence that makes us uniquely who we are was a concept reserved to science fiction. Today, that technology is very much a reality.


The question is, what do we do with it?


In the following article, we explore the capabilities of CRISPR, the current legal landscape and the future of this cutting-edge biotechnology.


The story so far: what is genome editing and CRISPR-Cas9?

On October 7th, 2020, the Nobel Prize in Chemistry was awarded to Jennifer Doudna and Emmanuelle Charpentier for their work in developing CRISPR gene editing. It was the long-awaited recognition they deserved for what the Nobel Prize Foundation called an “epoch-making experiment”, conducted back in 2012.


The experiment had unveiled a method for programming the naturally occurring CRISPR system – a type of immune response found in bacteria that acts like “molecular scissors” for cutting up DNA of an invading virus. Of course, it isn’t just viral DNA that can be edited. This breakthrough opened the doors to the potential of editing any DNA they desired, be it that of bacteria, plants, animals or humans.


Crispr-Cas9 is the second generation of technologies that opens the door to curing and preventing inherited genetic disorders, and the speed at which it is advancing means there are already a number of next-gen technologies waiting in the wings.  The promise that genome editing holds is vast: at present, it is being explored in research on a wide variety of diseases, including single-gene disorders such as cystic fibrosis, haemophilia and sickle cell disease.


However, scientists have further demonstrated its potential in treating and preventing complex illnesses such as cancer, mental illness, heart disease and HIV. Most of the changes are limited to somatic cells, and so cannot be passed on from one generation to the next. On the other hand, changes made directly to germline cells or in the genes of an embryo do offer this possibility – and this is the point at which ethical issues begin to arise.


If it is permissible to use this technology to prevent disease, where is the line drawn to prevent genome editing to be used in enhancing normal human traits? As often happens with disruptive technological breakthroughs, the more we accelerate in our efforts to advance the capabilities of this technology, the harder it is for legislators to keep up.



The first CRISPR babies

In December 2019, a red flag in this territory was raised when Chinese scientist He Jiankui was sentenced to jail for three years and fined 3 million yuan after he was found guilty of conducting “illegal medical practices.” His crime was playing a leading role in the research and subsequent creation of genetically edited babies.


The world of biogenetics flew into a frenzy of outrage, and understandably so -Jiankui and his collaborators had forged ethical review documents and misled doctors into unknowingly implanting gene edited embryos into two women.


A year earlier, Jiankui had announced at the International Summit on Human Genome Editing in Hong Kong that he had modified a key gene in several human embryos in a way that he thought could bring resistance to HIV. He further set out his intentions to spare the babies the risk of becoming infected with HIV in later life, arguing that such a technique could be pivotal in reducing the HIV disease burden in many parts of Africa.


The experiment was deemed premature, irresponsible, and unjustified because it exposed the babies to risks associated with gene editing for a benefit that could not be guaranteed.


He’s activities were described by The Chinese Academy of Sciences as being:


“a gross violation of both the Chinese regulations and the consensus reached by the international science community. We strongly condemn their actions as extremely irresponsible, both scientifically and ethically.”


Regulation of CRISPR genome editing

Today, the gene editing market continues to grow in demand from both the agriculture and healthcare sectors. However, such giant leaps in genome editing technology have naturally sparked discussions on the ethical and regulatory implications.


News that Jiankui had altered embryos couples during fertility treatment, leading to the birth of twin girls with modified DNA, was a clear signpost that regulatory frameworks to govern heritable gene editing is needed.


In response to the Jiankui scandal, the Chinese government did issue new regulations, which state that clinical gene editing research requires national approval. Then, in early 2019, the first American law was passed that regulates the use of CRISPR. Republican state senator, Ling Ling Chang, wrote a bill for California state law that prevents companies from selling CRISPR kits designed to modify human DNA.


In the UK, genome editing of human germline cells in research is regulated by the Human Fertilisation and Embryology Authority, and the use of human germline genome editing as part of IVF treatment in women is prohibited. However, as the science advances, gaps in the existing regulations will inevitably exposed.


A recurring roadblock in a globally joined-up approach to regulation of such practises is that heritable genome editing would result in outcomes that are at odds with principles of human rights.


Jennifer Doudna, who is known for her work on CRISPR-Cas9, says that, in less than 30 years, “it will … be possible to make … any kind of change to any kind of genome”.


Because of the potential of this technology, she and many other scientists and ethicists have called for a global moratorium on clinical uses of human germline editing. Now, International committees convened by the World Health Organization, the US National Academies of Medicine and Sciences, and the Royal Society are currently working to propose regulatory frameworks for doing clinical germline gene editing safely, if it is to be done at all.


Whether such a moratorium would be effective is not certain and is the subject of debate amongst the WHO and the research community. Equally as pressing is the question as to whether an international approach would facilitate a greater awareness of the moral challenges presented by germline editing and amplify a broad range of perspectives on the subject. It is also unclear how a global moratorium would be enforced and whether it would be supported by society at large.


Perhaps the first step would be to ensure germline editing studies for research-purposes only could be conducted safely to discover the true potential it holds – but as many scientists have pointed out, it’s hard to imagine that others like Jiankui wouldn’t follow in his footsteps and go rogue regardless.


The current consensus is clearly that germline editing for the sake of human enhancement is morally unacceptable. The lines between treating illness and enhancing human performance are too fine – something which has been demonstrated by doping scandals in the sporting world. There is a clear need for more research and discussion from voices across the board before genome editing can become a clinical reality.


Then there’s the challenge of financial disparity that persists in modern society. If genome editing becomes available primarily through the private sector, it becomes a tool that only the wealthy can take advantage of. This opens the floodgates to a broader debate about access to healthcare and what constitutes as urgent and necessary: if one family can afford to prevent genetic disease in their unborn child, such diseases would soon become a problem limited to those who are economically disadvantaged. This issue in particular is one that needs to be adequately addressed before genome editing can be considered ethically acceptable on society.


Many feel the first step would be for researchers to develop a system that allows early recognition of any research that is beginning to overstep the boundaries of health into human enhancement. A practical solution could be to follow the WHO’s guidance for regulating research with a biosecurity risk and putting in place a framework by which researchers could identify and signpost any research that could be viewed as dangerous to society.


The WHO’s Expert Committee on human genome editing draft framework for governance

Finally, in July 2020, the WHO Expert Committee issued a working draft framework for governance of human gene editing technology. In doing so, the framework addressed the role that genome editing has played in fighting the COVID-19 pandemic by assisting development of animal models to further our understanding of how humans experience the disease. However, the committee stressed that the draft framework is a work in progress; it emphasised that the road ahead in the scientific development of genome editing was long, and as such, the framework wouldn’t represent the final recommendations of the committee.

Nevertheless, the framework does signpost a step forward in the governance of genome editing and the use of CRISPR technology to cure and prevent disease in humans. It sets out some of the key considerations for robust governance of genome editing and a detailed assessment of the tools, processes and institutions currently available in overseeing the use of the technology.

Where do we go from here?

Before any official recommendations are made for policy development at both a national and international level, the WHO committee must excavate the extensive literature on the state of research. For a start, analysing what went wrong in the case of He Jiankui’s research and whether his experiments could have been prevented will help. From what we know, there were more than a few close associates of his who didn’t speak up – partly because they didn’t know whom to raise concerns to.


This flags a glaring gap that could be addressed: if such research was carried out again in a safe environment, scientists would need a mechanism for which they can raise the alarm if they feel the research risked going too far or was creeping into unethical territory. A change of practice would be necessary in the case of researchers who often choose not to intervene in the projects led by their peers.


Gene editing technology continues to evolve rapidly, and it raises serious issues that extend beyond fertility into ethical, social and economic considerations. Many countries such as the UK and the US, prohibit the use of germline genome editing in the creation of genetically modified humans.


Yet, many other nations still have no legislation preventing the use of germline editing and associated technologies. In the absence of such policy, there is a worrying potential of disparity between the approach of different countries. What one state considers unethical may be viewed by another as acting in the best interests of families and the population on a whole.


This reinforces the clear need for a robust governance framework in this rapidly evolving and fascinating branch of biotechnology. With great power comes great responsibility – if we are to take advantage of the potential that lies before us, continued collaboration on a global level is needed to develop and establish the rules that will allow us to harness this technology and put the brakes on before it goes too far.

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