A few days ago, a team of scientists from the University of Sichuan in China made a very important announcement, not only for the scientific community but also for humanity in general. In August this year, the researchers will use the gene-editing technique CRISPR-Cas9 (commonly known as CRISPR) for the first time in humans, in order to modify the genome of patients with lung cancer, for which traditional treatments have not been effective. But what is CRISPR and why this announcement should be of interest to all mankind?

The CRISPR technique is a method of synthetic biology that – for practical purposes in this article- acts as a sort of “molecular scissors”, which are able to locate, cut and replace specific sections of DNA, with a high degree of accuracy and control.

CRISPR origins date back to early nineties, following the publication of an investigation into an existing natural defense mechanism in bacteria that deactivates specific sections of DNA in viruses. In 2012, after years of having analyzed this immune system, the researchers Jennifer Doudna (USA) and Emmanuelle Charpentier (France), found that this mechanism could be replicated in virtually any other cell (including humans), in order to edit or deactivate very specific genes. The technique consists of introducing into a cell the enzyme Cas9 accompanied by a guide RNA. The enzyme makes a cut in a specific area of the target DNA, which sequences are complementary to the guide RNA sent, linking this new sequence and modifying the original DNA chain.

Unlike other gene-editing techniques, CRISPR seems relatively simple, accurate and versatile, which has opened the way for scientists to insert, modify, replace or delete sections of DNA for different purposes. And since DNA contains all the genetic information of living beings, handling it opens up endless possibilities. Practical examples of the use of this technique range from modifications of agricultural crops to improve their resistance to pests, to editing of specific human genes associated with genetic diseases, thus providing an alternative cure.

What is of great interest to all, is that CRISPR could also be used directly to modify the DNA in the germline (reproductive cells or embryos), which would mean that genetic modifications at this stage, would be inherited to future generations. While CRISPR represents a great opportunity to understand and treat many diseases, editing the germline would also open the possibility for discretionary “design” of “super-humans” with specific physical characteristics (eye color, height, etc.) or supernatural abilities (disease resistance, cognitive skills, etc.).

That is why in December 2015, a group of international leaders focused on this topic met in Washington to discuss the various ethical, legal and regulatory implications of making genetic modifications, mainly considering editions in humans and in the germline. So while the United States still expects to make its first human tests later this year, China announced a few days ago that it will begin with them this same month.

Following the discovery of CRISPR, we have seen the emergence of several companies that promise to deliver this editing technique. While CRISPR is still in an early stage of research and testing, several of these companies have already attracted the attention of major investors. The clearest example of this is Editas Medicine, a company co-founded by Doudna and other pioneers in the technique since 2013. The company raised $94 million before its initial public offering on February 2 this year.

CRISPR certainly is an extraordinary technological advancement and has the potential to revolutionize the world of medicine and biotechnology. It will be important to follow closely how these efforts unfold in this topic. There are those who dare to say that probably in the future, people will no longer die from disease or aging of their cells, but by circumstantial events or accidents.

NOTE: This article was originally published by Periódico Expreso on August 1st, 2016

Ricardo Celaya
Operating Partner at Break Off Capital
MBA Oxford University

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