Gene-edited Babies - The point of no return?
IT’S DONE! All predictions and speculations can be thrown out the window as He Jiankui announced the birth of the first gene-edited babies at the Second International Summit on Human Genome Editing in Hong Kong on 29 November 2018.
He Jiankui claims to have successfully implanted two embryos that have been edited with the CRISPR-Cas9 genome-editing tool (discovered in 2012 by Jennifer Doudna and Emmanuelle Charpentier) to disable the genetic CCR5 pathway. HIV uses CCR5 to enter and consequently infect human cells. The genetic change would make the gene-edited twin girls less vulnerable to HIV infection, not only at birth but for the rest of their life.
CRISPR-Cas9 (the technique used by He Jiankui) stands for Clustered Regularly Interspaced Short Palindromic Repeats, and it cuts the DNA. It was first discovered as an immune response of bacteria to protect themselves from invading viruses. By making a copy of the “foreign” DNA and making cuts, the CRISPR enzyme disables the genome of the invader. Nowadays, CRISPR-Cas9 can be engineered to target specific genes in the human genome and not only cut them, but repair existing errors (Find out more here).
With genetic editing a wide range of diseases are looking at potential solutions and even cures! The first clinical trials in China and the USA have been ongoing since 2016, and are showing great potential. There is however an important difference between the ongoing trials and what He Jiankui has done. So far, only ex vivo human experiments have been allowed to go into clinical trial. This means that a patient’s cells are taken out of the body (like immune cells), corrected to contain f.e. an extra gene to attack the cancer and afterwards put back into the patient.
The scientific community was shocked after He’s announcements, and even now many questions remain unanswered. Why did He Jiankui edit this specific gene while there are other strains of HIV using different pathways? Why use gene editing specifically for this disease, when there are many other ways to prevent transmission of HIV to the babies? Is the technique ready for this step? Do we understand and control off-target effects of CRISPR-Cas9 sufficiently? What about long-term effects on the twins?
While many of these questions do not have immediate answers, it is clear that He Jiankui acted against regulations released by China’s health and science ministries in 2003. On top of that, he is keeping the identity of the parents and babies a secret, making testing of He Jiankui’s claim by an independent body extremely difficult.
David Baltimore, Nobel prize winning biologist and chair of the summit where He Jiankui announced the news, criticises the lack of transparency and is calling He’s experiments irresponsible. Baltimore also admits a huge failure of self-regulation in the scientific community. Governments will now have to react, says Scot Gottlieb, the US Food and Drugs Association (FDA) commissioner. “The need for development of binding international consensus on setting limits for this kind of research, now being debated in Hong Kong, has never been more apparent.”, said Francis Collins of the US National Institute of Health (NIH).
Because of the recent and extremely quick developments in the field of genetics (f.e. decreased costs of sequencing, ease of editing techniques, faster and better read-out systems), gene editing has been the subject of many ethical debates and concerns worldwide. Unfortunately, thus far there are no clear rules or regulations about the use and implementation of this technique.
The majority of people would support genome editing in the context of curing diseases, but are against it for human enhancement purposes (intelligence, athletic ability), as shown by recent surveys. However, many other researchers along with myself fear that the efforts to use gene editing to help diseases could be hampered by He Jiankui’s actions.
The majority of people would support genome editing in the context of curing diseases
As Jennifer Doudna, one of the discoverers of CRISPR, and Samuel Sternberg say in their book The crack of creation: “This technique is the new power to control evolution!” How we want to implement it is up to us. Do we want to keep gene editing for uncurable diseases only? What about issues like aggression and restrictive mental conditions? Would chronic pain be okay to treat, or depression? Will we have a complete repertoire of designer babies gene-edited to our taste in the future?
Of course not all diseases are as simple as fixing one gene to eradicate the problem. Many diseases develop through a complex interplay between different genes and we are only just starting to reveal how different factors are involved.
The technique that sounds like science-fiction is here, and if we don’t want to lose out on its bright future, we NEED to implement and strictly enforce laws that will ensure safe and transparent research in the gene editing field. If we fail to do so, the bright future of gene editing may be lost in a sea of unregulated experiments that could harm participants as well as the public perception of this exciting technique.
How we plan to continue is up to us, ALL OF US. Gene editing is making its way to the clinic sooner then we think. Many of us might be confronted with gene editing in the future, so what will we do?
See also: Ukrant article - EXPERTS, current topics explained by RUG professionals.