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Human Genome Editing

Updated: Oct 20, 2019

By: Rashik Chand

There is no denying the fact that genetic engineering is a marvel of science and its advancement is indeed appreciable, however, with genetic engineering there is and probably always will be an associated ethical and social dilemma – pertaining to its use. With that being said, it is certainly possible to prevent a global outcry against genetic engineering by setting specific standards through consensus of the scientific community as a whole.

First of all, what is gene editing? Genome editing (also called gene editing) is a group of technologies that give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome. Several approaches to genome editing have been developed. A recent one is known as CRISPR-Cas9, which is short for Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated protein 9. CRISPR-Cas9 has created a wave of excitement in the scientific community and bring forth a new era of gene editing technology and associated research.

Human genome editing, as many scientists believe, holds the key to preventing and curing diseases that were once thought to be incurable. And on top of the list sits Cancer. In the race to find a cure for cancer, which, as per WHO, was responsible for 1 in every 6 deaths in 2018, scientists are optimistic that CRISPR is the answer. One of the first and most advanced CRISPR clinical trials is currently being run in China., testing the viability of genome editing in cases of advanced cancer of esophagus. Dr. Shixiu Wu, president of the Hangzhou Cancer Hospital heads the trial on immune cell therapy. The treatment starts with the extraction of T cells from the patient, which are then by using CRISPR are modified to remove the gene that encodes for a receptor called PD-1 that some tumors are able to bind to and instruct the immune system not to attack. The cells are then re-infused into the patient with a higher capacity to attack tumor cells. Dr. Wu claims that about 40 percent of the patients appear to have responded and is writing up the results for a scientific publication, but these results have not yet been peer-reviewed or published.

Further talking about what possibility genetic engineering holds; we cannot deny the fact that genetic engineering may possibly be our solution to preventing several fatal diseases. Most notably The individual—who is known as the ‘Berlin patient’—underwent two allogeneic haematopoietic stem-cell transplantation (HSCT) procedures using a donor with a homozygous mutation in the HIV coreceptor CCR5 (CCR5Δ32/Δ32) to treat his acute myeloid leukaemia; which lead to long-term HIV remission. Furthermore, the team lead by Ravindra K. Gupta, MA MPH MRCP PhD, in a paper published in the scientific journal “Nature” state that, “HIV-1 remission may be possible with a less aggressive and toxic approach. An adult infected with HIV-1 underwent allogeneic HSCT for Hodgkin’s lymphoma using cells from a CCR5Δ32/Δ32 donor. Antiretroviral therapy was interrupted 16 months after transplantation. HIV-1 remission has been maintained over a further 18 months.” The study proves that the cure of the “Berlin patient” wasn’t a one-off and additional research could possibly provide viable solution to the HIV epidemic.

Ethical concerns arise when genome editing is used to alter human genomes.The Nuffield Council on Bioethics said that changing the DNA of a human embryo could be “morally permissible” if it was in the future child’s interests and did not add to the kinds of inequalities that already divide society. The important point here is that is emphasizes on the fact that at present there is not enough research and safety procedures to ensure that the genome editing in the human embryo is successful. With that being said, there will always be the possibility with failure when speaking of scientific research or advancement but that is not something out of the ordinary, nevertheless, necessary precautions must be taken.

"My concern is: Are we really ready? There so much about CRISPR that we don't understand," says Lainie Ross, a bioethicist at the University of Chicago. "We could be doing more harm than benefit. We need to very, very cautious. This an incredibly powerful tool."

Adding on to that, at present most of the changes introduced with genome editing have been limited to somatic cells, which are cells other than egg and sperm cells. This is done so because, these changes affect only certain tissues and are not passed from one generation to the next. However, changes made to genes in germline cells (gametes such as the sperm or the egg are part of the germline) or in the genes of an embryo could be passed to future generations. Maybe in the future, with proper research and further scientific advancement it will be possible to edit germline cell in a controlled manner so as to, in principle, prevent children from inheriting serious diseases caused by faulty genes. Germline editing was always inevitable; with several scientists saying the same – for instance, Isaac Asimov, 20th century biochemist had said, “Genetic engineering just speeds up the process that used to take generations.”

It is not at all surprising that most people are not keen about using gene modification for making so- called “designer babies”. American biologist, Lee Silver had projected a dystopia in which a race of superior humans look down on those without genetic enhancements, though others have counseled against accepting this vision of the future. In November of 2018, the scientific community was shocked when a Chinese scientist, Dr. He Jiankui, announced that he had successfully edited the genes of twin girls to make them HIV resistant.  Though there are still a lot of questions surrounding this research and the actual results, and Dr. He’s actions have been largely condemned by scientists around the world, this has highlighted questions surrounding the ethics of manipulating the human genome and what the future of this type of science should look like.  It has also been suggested that if designer babies were created through genetic engineering, that this could have deleterious effects on the human gene pool. Some futurists claim that it would put the human species on a path to participant evolution. Again, another ethical question that is brings associated with it is who will have access to the technology - will it be only limited to the elite? It seems that only time will tell whether or not human genome editing is a blessing or a cure.



What are genome editing and CRISPR-Cas9? (National Institute of Health)

The Gene-Editing Conversation (American Scientist)


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