life (with) science

In 1997 a movie depicted a dystopian future of gene-edited people. Most of those technologies are now becoming real. What are they and what are the differences between that future and ours?

Jerome Morrow thinking: “The most unremarkable of events. Jerome Morrow, Navigator First class, is about to embark on a one-year manned mission to Titan, fourteenth moon of Saturn. A highly prestigious assignment, although for Jerome selection was virtually guaranteed at birth. He is blessed with all the gifts required for such an undertaking. A genetic quotient second to none. No, there is truly nothing remarkable about the progress of Jerome Morrow, except that I am not Jerome Morrow.”

This is one of the opening lines of the 1997 “Gattaca”, a sci-fi/biopunk movie directed by Andew Niccol. Gattaca (the name of which is made from the letters of the DNA bases: Adenine, Cytosine, Guanine and Thymine) shows a dystopian near future where the practice of genetic modification after in-vitro fertilization (IVF) is legally accepted. Therefore, future parents can easily go to the “local geneticist”, select the best characters for their children like I.Q., skin, hair, and avoid genetically based illnesses or disorders as well as negatively perceived traits like premature baldness or myopia. The result is a society where naturally conceived children are discriminated against genetically modified ones and segregated. At least until a man called Vincent, whose surname couldn’t be anything but Freeman, succeeds in pretending to be a member of this elite of gene-edited people. Do these technologies really sound like science-fiction anymore?

As I explained in my previous posts, gene-editing techniques are growing and spreading faster than ever, and new applications of them are frequently published. There are three main gene editing techniques available today: TALENs, zinc-finger nuclease (ZFN) and CRISPR-Cas9, the last of which appears to be considerably easier to perform than the former two. The international scientific community as well as the general public are getting more interested in this new field of biology, especially after the first study concerning genetic modification on human embryos was published earlier this year.

Many scientists think that it was just a matter of time; nevertheless when on April 18 2015 the online journal Protein & Cell published a Chinese study on gene-edited human embryos, both the scientific community and the public opinion expressed concern. The debate on how to use such powerful techniques was on the top of scientists’ priorities since the beginning of the year when a meeting took place at Napa, California to discuss the scientific, medical, legal, and ethical implications of these new technologies. Nature’s news reported that there were leaks at the beginning of the year about this and other similar studies by several researchers who preferred to stay unnamed. Moreover it is interesting to notice that both Nature and Science refused to publish the study. So what is this study about?

At the Sun Yat­sen University in Guangzhou, a team led by Junjiu Huang attempted to modify the HBB gene responsible for β­thalassemia, a potentially fatal blood disorder, using CRISPR-Cas9 technique. Huang and his colleagues were fully aware of how delicate and controversial their study was, therefore they used non­viable embryos they took from a local fertility clinics; that means embryos that due to severe chromosomal deletions or duplications cannot result in a live birth, in other words, a waste product. Contrary to what many people could think, the results revealed serious obstacles in using the method for medical applications. Of 86 treated embryos, only 28 were successfully edited by CRISPR-Cas9 enzyme and only a few of the

se embryos contained the replacement genetic material. Moreover, the team found an unexpectedly high number of off-target mutations, which means that CRISPR-Cas9 acted not only on the specific HBB gene, but also on several other regions of the genetic code that were not supposed to be targeted. This is surely surprising for a system like CRISPR-Cas9, which is considered to be highly efficient and specific. “If you want to do it in normal embryos, you need to be close to 100%,” said Huang.

The case inevitably raises several ethical questions and creates what in law is called “the precedent”. Is it ethical to use human embryos even just non-viable ones, in order to understand the system more deeply? There are many inheritable diseases or predispositions that could be finally treated using these powerful tools. Therefore, where is the line between what is ethical and what is unethical? How can the application of these techniques be strictly regulated world wide in order to allow their use for severe conditions and prohibit it for the creation of what are journalistically called “designer babies”?

These and many others are the ethical issues that societies and the scientific community are facing now. So if the dystopian world of Vincent Freeman/Jerome Morrow is undoubtedly unrealistic, the main question raised by this eighteen years old movie are now more actual than ever. What in 1997 were futuristic technologies are now a reality; we are now (partially) living that future. I think that we are all involved in this matter because such technologies will certainly become a part of our daily life. “The question is when, not if”.

I will tackle these and many other questions during two other posts, using Gattaca as a tool for helping readers to understand such a difficult, but extremely important matter.