The ability to edit genes in living organisms offers the possibility of treating a multitude of hereditary diseases. However, many types of gene editing tools are not able to reach critical areas of DNA, such as the noncoding region. But a new technology may have taken a new step in that direction.
The new procedure called SATI was developed by the Salk Institute for Biological Studies, a scientific research center located in the United States, and was tested in a report recently published in the journal Cell Research.
DNA-modifying techniques, particularly the CRISPR-Cas9 system, have a higher level of efficiency in dividing cells, such as those in the skin or bowel, using normal cell repair mechanisms. But now, as the study co-author explains, research has attempted to "create a versatile tool for targeting the non-coding region of DNA, which would not affect gene function and allow the targeting of a wide range of mutations and types of DNA." cells ".
The noncoding region of DNA contains no "instructions" to make proteins and makes up the bulk of the total genome size, which integrates the genetic code with hereditary information from an organism. Therefore, this technology can be very valuable for future therapies. To prove this potential, Mako Yamamoto explains that the research focused on "a model of premature aging caused by a mutation that is difficult to repair with current genome editing tools," the progeria syndrome.
Technology that altered the genome allowed the team to detect a wide variety of mutations and cell types. According to the scientists, this system could be expanded to a wide range of genetic mutation conditions in humans, such as Huntington's disease, an inherited brain disorder, and a rare disease that causes premature aging, progeria.
Scientists tested the technology in living progeria mice, caused by a mutation in the LMNA gene, and once applied, the researchers noted aging regressions in various tissues, such as skin, along with a 45% lifespan, compared to rats to which this process was not applied.