Editing a person's DNA to fight or prevent various diseases is a matter of the near future. Encouraging results of application not only in animals, but also in humans, of CRISPR technology show that the revolution in medicine is very close.
The method for editing the genome of CRISPR appeared only in 2012, but proved to be so productive and effective that it was already begun to be tested not only on animals (one of the last examples is the cure of mice from glaucoma), but also in public. About 20 experiments involving human patients began or should begin soon. Almost all of them are associated with editing DNA extracted from the body. It is relatively easy to remove immune or stem cells and return them back after correction, however, with most tissues it is impossible to do so.
Only editing cells inside the body will treat most diseases - from genetic disorders to elevated cholesterol - and will be cheaper than growing and editing cells outside the body. When asked what diseases can be treated in this way, Irina Konboy of the University of California at Berkeley responds: "Absolutely everything".
The main problem of editing tissues inside the body is the method of delivering CRISPR technology inside the body. This requires at least two components: a protein that cuts the DNA, and part of the RNA that will direct it to the exact place in the DNA to be cut. Compared to conventional drug molecules, proteins and RNA are huge, and it is difficult to deliver them to the cells, in addition, they usually do not experience travel through the circulatory system.
However, biologists have been working on this task for many years and with the advent of CRISPR they are ready to share their experience with geneticists.
For example, the American company Intellia Therapeutics uses fat particles to deliver CRISPR components to the liver. Last week, her specialists were able in this way to disconnect the mouse in the mouse, the TTR gene, responsible for the production of proteins of transthiretins that cause systemic amyloidosis. And the team Conboy managed to perform even more difficult task - not to disconnect, and fix the damaged gene, to restore muscle function in a patient suffering from dystrophy, according to Haytek, citing New Scientist.