The gene editing technology notoriously known as CRISPR/Cas9 has gained vast attention after the Chinese scientist He Jiankui claimed he had, for the first time ever, created gene-edited twins. While He has received negative criticism for his work amongst the scientific community, many believe the world can benefit from this powerful technology. He Jiankui has decided that the world is ready for germline gene editing, but does the rest of the world feel the same?
Why the outrage?
By now, every news channel has covered the controversial story regarding the Chinese scientist He Jiankui, who claimed he created, for the first time ever, gene edited babies named Lulu and Nana using the gene-editing technology known as CRISPR/Cas9. His work was presented at a summit in Hong Kong in November 2018 before being peer-reviewed or published in a journal, which raised eyebrows within the scientific community. He has also failed to share the detailed scientific methodology used in his study, confusing scientists further, as this is of utmost importance when deciding whether any experiment can be deemed successful or whether its scientific breakthroughs are even true. For scientists across the globe, this was a clear example of scientific misconduct, carried under unethical standards with very little transparency between the scientist and the public.
What He has done is simply irresponsible and has caused a severe backlash from scientists, although his intentions seem to have come from a good place, mainly driven by the desire to protect these children from contracting AIDS. He Jiankui believes that by deactivating the CCR5 gene, a gene that has previously been shown to play a big role in HIV infection, these kids would be immune to the disease. However, people with CCR5 mutations also risk contracting other infections. Since Lulu and Nana did not have HIV in the first place and there are several anti-CCR5 drugs in the market, many raise questions on why He was so fixated on such drastic experimentation. Nevertheless, gene-editing in the germ-line is not just risky, but is currently illegal in China (1) and He never tested what this mutation would do in immune cells or mice prior to rushing into germ-line editing into humans. Unsurprisingly, He has disappeared, but what has been left behind is a negative stigma surrounding CRISPR/Cas9 by the general population, which does not fully understand this powerful technique and its alternative applications.
He Jiankui for worst scientist of the year
If a bad scientist of the year award existed, He Jiankui would be the top pick. It boils down to this: there is no such thing as a perfect scientist. Nevertheless, there are three basic qualities that scientists require, defining what it takes to be at least a decent one. These include: 1) solve a real problem, 2) bring results to light with sound experimental plans and 3) have complete transparency of your results with the scientific community and especially with the public. Having the right intention is rather biased, as He truly believes he is doing the right thing; however, his experiments have left the entire scientific community questioning “what was the purpose?” “why has he done illegal experiments?” and “why was everyone left in the dark about this research?”
Clearly, not scientist of the year material.
CRISPR what?
To better understand the matter at hand we must first understand the complexity of CRISPR/Cas9 technology by making the science behind it as understandable as possible. Sometimes scientists get carried away with complex terminology. This not only leads to confusion within the scientific community but also within the general public, which does not fully understand what occurs in a research laboratory, and most importantly, the scientists’ ultimate goals.
CRISPR/Cas9 was discovered not too long ago to be the immune system that bacteria and archaea use to ward off viruses. The term stands for short for Clustered Regularly Interspaced Short Palindromic Repeats, or rather a sequence of complex words that mean absolutely nothing to most of us. In simplified terms, they are basically small gene fragments belonging to phages (viruses that attack bacteria) that the bacteria store up to remember what the virus looked like, a viral fingerprint one could say. When a similar phage attacks the bacteria again in future, the Cas9 enzyme (nothing more than molecular scissors) can recognize these small genetic sequences and slice through them leading to phage killing (see Figure 1). Ultimately, this ‘cut and replace’ system works by cutting away viral DNA and replacing it with bacterial DNA. It is fascinating to know that even bacteria and archaea have developed their own “immune system” and scientists are now using this tool to exploit new therapies and enhancements in the agricultural and medical fields.
Now that the technology has been explained we can move on to why He’s work seems so scandalous. If his findings are indeed true, Nana and Lulu could create gene-edited children that could negatively impact the genomic profile of individuals in future generations. The potential to do good is enormous: CRISPR can potentially be used to eliminate a genetic disease from a family forever, switch or replace genes to improve plant growth and treat animals for deadly infections and diseases. Thanks to gene editing, this technology is being implemented to replace pesticides for sustainable crop growth which would also benefit the environment. Additionally, the technology has been used to treat several medical conditions such as blindness and cystic fibrosis, but if this ‘cut and replace’ system goes wrong, the consequences are potentially eternal, affecting future generations who could not give prior consent or in the worst situation, introduce life-threatening mutations. The ethical and safety concerns surrounding gene editing are clearly outstanding, and for this reason the International Summit on Human Gene Editing was created in 2015 in Washington, DC. Led by the United States, United Kingdom, and China, its main goal is to harmonize regulation of genome editing technologies. Although gene editing has been deemed safe by the NIH, the benefits don’t seem to outweigh the risks when using human embryos, since other technologies like the PGD (Preimplantation Genetic Diagnosis) are performed during IVF treatments to allow detection of faulty genes that could lead to disease. Some scientists also worry that germline editing would also encourage baby enhancements for non-medical reasons i.e. designer babies. Whilst other researchers like He strongly believe the world will only benefit from gene editing technologies in the germ-line, many moral philosophers argue otherwise. One thing is certain about He’s actions: before we could even think of performing germ-line editing, a tremendous amount of preliminary research was necessary before He could go anywhere near an embryo. The world was not ready for CRISPR babies.
References:
- National Academies of Sciences, Human Genome Editing: Science, Ethics, and Governance, The National Academies Press, 2017.
Received: 05.01.2019, Ready: 30.01.2019, Editors: ST, RG.
