For the first time, the prize goes to two women, Emmanuelle Charpentier and Jennifer A. Doudna, for the CRISPR technique

Their CRISPR/Cas9 DNA editing technique opened up to unexpected prospects, for the most part still unexplored, in terms of editing, never before so fast and accurate, of genetic defects. 

Because they were able to “rewrite the code of life” - therefore paving the way to unimaginable and impossible genetic therapies - the 2020 Nobel prize for chemistry was awarded to Emmanuelle Charpentier and Jennifer A. Doudna. 

Theirs was a double victory: for the first time a scientific Nobel prize was awarded to two women. Since its institution in 1901 to today, the Nobel prize for chemistry was awarded to five women only.

A French biochemist the former, an American chemist the latter, almost ten years ago Charpentier and Doudna designed - their study was published in 2012 - a method which is now a reference point to edit the DNA of plants and animals. The potential applications on human DNA deserve a separate discussion.

In 2018, the two researchers were among the candidates to the “Lombardia è Ricerca” international award of Regione Lombardia: their names were put forward by VIA - Top Italian Scientists members, which are asked every year by the Region to indicate the most significant Life Sciences discovery for the subject provided from time to time as the Award focus.  

“It is with great pride that we have learnt about the Nobel prize to two researchers who, by studying DNA, are allowing to understand and cure many diseases - commented the Vice president of Regione Lombardia and Councillor for Research, Innovation, University, Export and Internationalisation Fabrizio Sala -. Regione Lombardia has allocated over one million EUR for research programmes on human genome sequencing to enhance the ability to prevent diseases. This work is fundamental - and the Nobel prize is evidence of this - and we intend to be ready to tackle our future challenges with precision medicine, which happens to be a priority of our three-year strategic plan for Research and Innovation”.

The technique

The discovery of the two researchers may be defined as a pair of “molecular scissors”, activated by a protein, Cas9: Indeed, Charpentier and Doudna - whose research collaboration started in 2009 - discovered how to leverage the ability to program this protein to perform specific changes to the genome of a cell - be it animal, plant or human. They registered several patents based on this technique.

More specifically, the research groups led by the two scholars were first to show that this technology - borrowed from bacteria - can be deployed as a biotechnological tool to edit the genome of a non-bacterial cell.

Thanks to the CRISPR/Cas9 method, it is possible to get rid of damaging DNA sequences from the target genome, or replace other sequences, thus correcting some mutations that have been identified as the cause of specific pathologies. Hence the description as a DNA “cut and paste” technique. 

All this with extreme accuracy and in fast time, while prior to the introduction of the CRISPR/Cas9 method, editing the genes in a cell was very difficult, the results were not reliable and the time required was much longer. 

Potential uses

The molecular scissor system designed by the two scholars opened the way to a number of therapies. It has already been used against a number of hereditary diseases, while on another front, researchers are testing its use to modify immune cells, in order to enhance their efficacy in their reaction to cancer cells. 

The results obtained are still partial, and on several occasions the technique has proven that it must still be perfected. Most importantly, several people raised the issue of the potential implications of abusing the CRISPR/Cas9 method: base research can benefit enormously from it, but for the first time the opportunity to create genetically-modified humans or to produce irreversible changes to the human gene pool is on the horizon. 

Biographies

Charpentier, 52 years old, is head of Berlin’s Max Planck Unit Institute for Pathogen Science, while Doudna (56 years old) after her studies at Harvard University moved to the University of California, Berkeley, where she currently works.