In 2006, a case of a rather rare genetic disease was described. One boy, originally from Pakistan, showed a very interesting deviation: he could feel the warmth, cold, texture of objects, easily recognize touch, but did not feel pain. Unfortunately, this boy is no longer alive, since he believed in his “invulnerability” so much that he jumped from the roof in an attempt to prove it. However, a group of experts managed to conduct some research on the genome of a young Pakistani, and these data today will help to createpainkillers that will act at the genetic level. They will work better than opiates and will not be addictive.

How to relieve pain

As you know, in order to develop such an anesthetic, scientists had to use the CRISPR genome editor . A new approach to pain relief imitates a rare mutation of a Pakistani boy. And he’s already tested on mice in the Prashant Mali lab at the University of California at San Diego. 

A study in which CRISPR was usedto temporarily block the pain, it took place in July, but the scientists did not want to give any comments until all the data of their research passed a full check. The main idea is to introduce virus particles into the cerebrospinal fluid that carry a modified version of CRISPR designed to interrupt pain signals. Modification (or rather temporary blocking) is subjected to a gene called SCN9A, which is responsible for the production of a particular molecule (it was called Nav1.7). It, in turn, is a key factor in the passage of a pain impulse into the brain.

This is interesting: Using CRISPR, for the first time, it was possible to edit several genes simultaneously.

Evidence of the dominant role of this gene and its corresponding molecule was obtained from people with strange hereditary diseases that cause a decrease in the pain threshold. The Pakistani boy already mentioned at the beginning of the article was a member of a family with a mutation that completely turned off the SCN9A gene. Thanks to, it was possible to study this mutation quite well. But so far, attempts to mimic this mutation have not been successful, but researchers from California have succeeded.

The data obtained during experiments on animals suggest that it is not yet possible to completely remove the pain syndrome. But to significantly reduce its manifestation is already quite capable. The effect of CRISPR therapy is comparable to the use of opiates. The only exception is that CRISPR is not addictive. This is a huge step forward.

Innovation: CRISPR Gene Therapy Pain Relieves Better than Opiates
Computer simulation of how CRISPR works when DNA fragments are “cut out”

At the moment, scientists plan to start full-fledged research involving people. Experts say that such gene therapy will help to relieve a huge number of people from chronic pain. For example, patients in advanced stages of cancer or people with congenital neuralgia. Also, one cannot exclude the more “standard” use of a new type of pain medication: the use of CRISPR drugs during operations or in patients with serious injuries, for example, after accidents or in war zones. 

For faster entry into the market, scientists have created the company Navega Therapeutics, which will work with the FDA (U.S. Food and Drug Administration) and a number of research centers to test the health of the new drug.