The notorious CRISPR-Cas9 editor has become a real breakthrough in genetics. With his help, many discoveries were made. He also laid the foundation for such a phenomenon as "gene therapy". However, a group of physicians from the University of California, San Francisco, in collaboration with scientists at the University of California, Berkeley, are planning to go further and use the CRISPR editor as a "genomic surgery" tool to heal people from rare genetic diseases.
The fact is that various genetic defects can be quite rare and not so many people get sick with them. This means that, unfortunately, it is not profitable to develop drugs for the treatment of these pathologies. Of course, developments in this area have not been completely stopped, but due to lack of funding they are going "slower than we would like." And this is where a genome editor comes in handy.
Scientists are now working with cells that are affected by diseases such as vitelimorphic dystrophy and Charcot-Marie-Tooth disease. The first is a disorder of the visual system that causes gradual loss of vision, and the second is characterized by muscle atrophy of the limbs, neuropathy, and a number of other symptoms. But the authors chose these diseases not for this reason, but because they are rather “easy targets”, as they are caused by single nucleotide substitutions in one gene. Therefore, it is quite easy to develop treatments. Now scientists are working out various options for "genomic surgery" and if their experiments are successful, we can expect the development of methods of treatment and more serious diseases.
Vladimir Kuznetsov
