The notorious editor of the CRISPR-Cas9 was a breakthrough in genetics. With his help I made a few discoveries. It also marked the beginning of the phenomenon of “gene therapy”. However, the group of doctors from the University of California San Francisco in collaboration with scientists at the University of California at Berkeley are planning to go ahead and start using the editor CRISPR as a tool for “genomic surgery” in order to cure people from rare genetic diseases.
The fact that different genetic defects can be quite rare and they hurt so many people. This means that, unfortunately, to develop drugs to treat these pathologies unprofitable. Of course, development in this area is not completely stopped, but due to lack of funding going “slower than we would like.” And it is here that can be useful to the editor of the genome.
“Imagine the following scene: people go to the doctor, where he sequanorum their genome, then they will know that they have a genetic disorder. And instead of telling them what they need to live with this disorder, we have technology that can actually cure them.” — say the authors work.
Now scientists working with cells that have been devastated by diseases such as vitamina degeneration and disease Charcot-Marie-tooth. The first is a disorder of the visual system that causes a gradual loss of vision, and the second is characterized by atrophy of the muscles of the limbs, neuropathy and other symptoms. But the authors chose those diseases not so, but because they are “easy targets”, as caused by single nucleotide substitutions in a single gene. Therefore, to develop methods of treatment is quite simple. Now scientists are working out different options, “genome surgery” and if their experiments succeed — we can expect the development of methods for the treatment of more serious diseases.
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