CRISPR from bench to bedside

The progress of the new therapies of the CRISPR era can be told by interweaving two stories. The first is the one featuring Victoria, Carlene, Patrick, Alyssa, Terry and many others. There are over two hundred patients who have so far undergone some experimental treatment based on genome editing, i.e. the targeted correction of DNA instead of the addition of extra genes as in classical gene therapy. These women and men suffering from serious diseases had to face increasing pain and sacrifice until they decided to pin their hopes on a new type of experimental therapy, which is promising but not without risks. For the unluckiest of them, this act of courage and faith in science was not enough, but for many of these pioneers, life really did change. In fact, there are already dozens of people who have managed to free themselves (hopefully in the long term) from the burden of a rare genetic disease or, in some cases, leukaemia. Along with genetically edited cells, a new normalcy has arrived for them and the chance to finally think about the future.

The second narrative thread is the one woven by the scientists who invented, perfected and tested the biotech tricks on which these advanced therapies are based. The best-known protagonists of this adventure of knowledge are Jennifer Doudna (University of California at Berkeley) and Emmanuelle Charpentier (Max Planck Institute in Berlin). It is they who in 2012 invented the CRISPR genetic scissors, capable of modifying selected DNA sequences at will. The acceleration that this tool gave to research in biology and medicine earned the two researchers the Nobel Prize in Chemistry. But in the space of a decade, such a large and vibrant scientific community has grown up around this biotech innovation that it has become impossible to pay tribute to all those who, in the public and private sectors, are helping to bring CRISPR from the bench to the bedside. (This is the lead of the 6 pages feature I wrote for the Italian edition of Scientific American)

Leave a comment