Amplifier instead of scissors
Gene activation instantly coped with obesity
Svetlana Maslova, Hi-tech+
Genetic disorders in obesity can be corrected without editing the genome. Scientists from the USA have developed a new technology for strengthening CRISPRa genes (a – activation), in which DNA does not need to be cut.
Researchers from the University of California at San Francisco (USA) proved that such CRISPR therapy copes with genetic defects of obesity without DNA cuts. Their new technology called CRISPRa does not use genome editing, but only enhances the activity of a copy of the gene. And it demonstrates excellent results, scientists say in a press release CRISPR Joins Battle of the Bulge, Fights Obesity Without Edits to Genome.
It is known that the human genome contains two copies of the gene – one from each of the parents. At the same time, there are at least 660 genes where a mutation in just one copy leads to a disease. Obesity is among them.
Mutations in SIM1 or MC4R – two critically important genes for regulating hunger and satiety – are more common in obese people. When one copy mutates, the body relies entirely on the second. But often it does not sufficiently inform the brain about saturation. As a result, a person consumes food uncontrollably, and he develops obesity.
In an experiment with mice, scientists found a functional copy of the gene, strengthened it – and achieved excellent results. Mice without one of the copies of the SIM1 gene received CRISPRa injections at the age of four weeks and were able to maintain a healthy body weight. Without treatment, the rodents ate constantly and suffered from obesity at the age of 10 weeks.
Experience has shown that even a single CRISPRa therapy fixes the result for a very long time.
The observations were carried out for ten months – this is a significant period of life for rodents, the authors specify.
In addition to obesity therapy, CRISPRa can also be used to adjust the "volume" of genes in many other diseases caused by the weakening or defect of one copy. The method demonstrates great therapeutic potential for a number of diseases.
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