I recently watched an episode of one of my favourite TV shows, the Good Doctor. The episode revolved around how a man named Wyatt was rushed into the ER as he had a perforated valve and needed immediate surgery. As the doctors were operating on him, they noticed diffused dilation of the large intestine, calcified lesions and thinning of the intestinal wall.
These are all symptoms of Hirschsprung’s disease, a rare genetic defect observed in babies and young children. The question now was how a man in his mid-30s acquired a disease which only affects toddlers. Upon interrogation, it was revealed that Wyatt had modified his genetic code via a CRISPR procedure under the counselling of some professionals he knew in China. They sent him pharmaceutical ‘cocktails’, which he then injected into his bone marrow, all whilst believing that this would stop him from ageing and allow him to live forever.
Wyatt wanted to be ‘immortal’ and was ready to risk it all by altering his genome, i.e., the very component of his body that distinguishes him from the rest of the world. Moreover, once this experiment proved successful for him, his wife intended to get the same procedure done to her. All of this seemed rather bizarre to me. The fact that Wyatt was ready to trust people he barely knew through work and that he was ready to do a life-altering procedure to stop something as natural as ageing seemed, well, peculiar.
What Is Human Gene Editing
Human gene editing is a technique that allows the deliberate alteration of a human’s DNA in order to modify specific genes. This procedure can also add or delete certain parts of our DNA.
Genes are arguably one of the most crucial facets of the human body. Genes are what make us humans. They regulate our hair type, eye colour, height and even personality.
Any change in our genes could have a profound impact on the human species as a whole. Even the alteration of one nitrogenous base in the DNA sequence, something as minor as the flipping of adenine and thymine nucleotides, could cause mutations and adverse effects thereafter.
Therefore, any changes that alter the genetic code of any organism must be done with extreme precision. Gene editing is done by using enzymes, specifically nucleases, engineered to target certain sequences of the DNA strand and make necessary changes.
Gene editing is rather sui generis, with the molecular tool CRISPR (short for clustered regularly interspaced short palindromic repeats) discovered in 2012 by American scientist Jennifer Doudna and French scientist Emmanuelle Charpentier.
The Invention Of Technique
The idea to use gene therapy to treat congenital diseases dates back to when Watson and Crick discovered the double helix model of DNA in 1953, and subsequently, researchers realized that even minute changes in genes, passed from parents to their offspring, could cause mutations and diseases.
Identification followed by rectification of these molecular mistakes is the only way to cure these diseases. The development of recombinant DNA (rDNA) technology in the 1970s, where restriction enzymes were used to ligate specific strands of DNA, was a significant breakthrough in this field.
In 1990, scientists treated a patient with ADA-SCID, an infrequent genetic disorder. ADA-SCID, more commonly known as ‘bubble boy disease’, is the most severe form of immunodeficiency, which causes lymphocytes to die.
Simply put, lymphocytes are cells in our blood that provide immune response and fight various pathogens. The patient’s immune system becomes excessively susceptible to various communicable and air-borne diseases in SCID. This is why patients with this disease are often surrounded by a plastic ‘bubble’ containing sterilized air that acts as a mechanical barrier between them and several pathogens. In 1994, zinc finger nucleases (ZFNs) were engineered and became the first customizable tool that enabled targeted modifications in DNA. Other technologies like TALEN and CRISPR-Cas9 make gene editing affordable and improve their efficacy. In 2018, He Jiankui, a Chinese scientist, edited the genomes of twin female foetuses to make them resistant to HIV. They were later delivered successfully.
If we talk about the scope of gene editing, it can have widespread uses in medicine, agriculture, animal breeding, industrial biotechnology and the environment. In medicine, it can be used to treat monogenic illnesses (diseases caused by mutation in a single gene) like cystic fibrosis, sickle cell anaemia and Duchenne muscular dystrophy. It can also be used to treat cancer by stimulating the immune system to initiate responses that attack cancer cells (CAR-T cell therapy). In agriculture, it can improve crop traits and enhance nutritional impact. It may also be used to reduce the harvest period of commercial crops alongside techniques like vernalization. In animal breeding, gene editing can enhance the yield of animal products and increase the tolerance of animals to diseases. Genetically engineered microbes can be used to clean contaminated areas through bioremediation.
If gene therapy has so many benefits, then why has it not been implemented everywhere? Apart from the overall novelty of this procedure and its success rate, the main issue with gene therapy is that many people think it is unethical.
To understand the ethical dilemma, I conducted a survey with most of the participants belonging to the 11-20 and 31-40 age groups. 46.8% believed that human gene editing should be legalized in developed countries like the USA, Russia, China and Japan, while at the same time, 36.2% were against its implementation in underdeveloped countries due to concerns regarding the exploitation of biomedical data and biopiracy.
A majority of the participants wanted gene therapy to be used for the treatment of mental illnesses and neurodevelopmental disorders like ASD (autism spectrum disorder) and help with neurotransmitter regulation. 57.4% believed gene editing would further widen the socioeconomic divide and be affordable to only a tiny fraction of the population.
Yet again, this would be another tool practically inaccessible to the masses. 80% of the participants thought that gene editing would make society less accepting of human errors and would uproot what makes us humans in the first place- our inadequacies. There was a 50-50 divide regarding whether humans have reached a stage where procedures like gene therapy and human principles like empathy and compassion can co-exist. It must also be noted that gene therapy is effective only on somatic (body) cells and not germ cells (sperms and ova).
This implies that the edited gene would be valid only for one generation and not be inherited by the progeny. On further analysis, the issue is not actually regarding human gene editing itself but the fear that the legal and medical framework would not be cohesive enough to prevent malpractices.
For instance, human germline editing where embryos are genetically altered to create perfect, intelligent, healthy and well-balanced individuals or ‘designer’ babies. Many were against elective surgeries, which were focused on improving or perfecting skills rather than curing illnesses. According to some, human gene editing does not resonate with principles like morality and empathy, which a person develops through experiences, relationships and fighting through difficult circumstances. Furthermore, there is the risk of unintended immune reactions and mutations, where the immune system rejects the new genes and initiates cell autolysis.
It would be challenging to gain consensus of different countries and agree to specific regulations. This lack of uniformity on a global scale could further widen this predicament and also lead to regulatory loopholes, given that countries like Belarus, Canada, Switzerland, and Sweden have already banned germline editing trials.
Although successful attempts have been made on animals, where scientists injected jellyfish DNA into cats and discovered that they could glow in the dark, is technology really advanced enough to carry out this procedure on humans? Have we as a society progressed enough to reach an egalitarian stage where we can accept various kinds of people, be it humans with defects or robots with no emotions? Is human gene editing just a desperate attempt to eradicate all our flaws and imperfections? Is it ethical to use gene therapy on an embryo even though it is literally impossible to get permission from the embryo for treatment? Is it alright for people to use gene therapy to increase their height or improve their athletic ability when thousands of
others are in dire need of help? Should scientists even be allowed to experiment on germline editing? All these are valid questions which need to be thought of before considering the legalization of human gene therapy.
While many of us are hopeful for the future of gene therapy in the medical and agricultural field, others are still dubious about facets like regulation, economic stratification, treatment delivery and the procedure’s long-term viability. The possibilities are immense, so are the risks. It is essential to balance technology with ethical considerations and harness the power of gene editing to benefit society as a whole.
(Ishana is a grade 11 student. This is her original article)
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