Director Iain Mattaj commented the winners of the 2020 Nobel Prize for Chemistry: “The Nobel prize awarded to Charpentier and Doudna is very well deserved. CRISPR is a typical example of a discovery which initially seemed interesting to a limited, niche group of scientists studying bacterial evolution. It has since become a fundamental tool for biology and subsequently an experimental tool for therapies linked to human health.
Genetic engineering, first achieved in 1972, is the ability to manipulate the structure and composition of DNA molecules by “cutting and pasting". It allows us to learn what genes do, how they work and why they go wrong.
CRISPR technology has the potential to make the “dream” of genetic engineering come true: to make specific, targeted changes in the genome of any organism.
Since the breakthrough of Charpentier and Doudna, thousands of experiments have been published showing that CRISPR systems work to enable “cut and paste” experiments in the genomes of a large variety of organisms, including human cells and organoids in culture. Off-target side effects are limited and can be measured and controlled. This progress is already enabling clinical trials of the use of CRISPR in certain types of cancer treatment. Once its safety is established, the method will be applicable to many genetic disease states, and also potentially to treat parasites that infect or damage humans, like viruses.
Meanwhile, researchers are applying the method to a wide variety of questions. At Human Technopole for example, we are using it to help understand neurodevelopmental disorders, to identify promising drug targets in different cancers and to understand aspects of the evolution of the brain.”
Image credit: Nobel Media. Ill. Niklas Elmehed.