One study suggests that a new genetic editing tool could give hope to people suffering from HD and progeria that lead to premature aging.
Study published in Cell Research indicates that researchers at Salk Institute have developed a new tool "This study has shown that SATI is a powerful tool for editing the genome," said Juan Carlos Izpisua Belmonte. Professor in the gene expression laboratory of Salk and lead author of the study in a statement. "It may prove useful in developing effective strategies for the replacement of many different types of mutations by target genes and opens the door to the use of gene editing tools to potentially cure a wide range of genetic diseases."
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Instead of completely replacing the disorder genes, the researchers used SATI to insert a good copy of one of the genes into a specific portion of the mouse's DNA. The gene is later inserted into the genome and essentially corrects the mutation without replacing the entire gene.
In the tests, scientists used SATI to correct the gene responsible for progeria in mice. The researchers said they observed reduced aging in multiple organs, including the skin and spleen, as well as a 45 percent longer lifespan of the mice with the disease.
"We wanted to develop a versatile tool to target these non-coding regions of DNA that would not compromise gene function and target a wide range of mutations and cell types," added Mako Yamamoto, one of the co-authors of the study. added. "As a proof-of-concept, we focused on a mouse model of premature aging caused by a mutation that is difficult to repair with existing genome editing tools." Huntington Disease Society of America. The genetic disorder "causes the progressive breakdown of nerve cells in the brain" and impairs the physical and mental abilities of a person in the first years of life. Currently no cure is known.
A spokesman for the Salk Institute told Fox News: "Huntington is cited as an example of the type of mutation to which this approach could potentially be applied, but mice with Huntington's were not involved and further investigations would do so for the Application of SATI technology to this specific disease may be required. "
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SATI is based on the CRISPR-Cas9 gene editing technology and works with a wide range of gene editing tools. George Church, professor of genetics at Harvard Medical School, is one of the inventors of CRISPR-Cas9.
"A similar lifetime extension, if applied to humans, would take more than a decade," the statement said. "Thus, the SATI system represents the first in vivo gene correction technology targeting non-coding DNA regions in multiple tissue types."
Later, the team aims to make SATI more efficient by increasing the number of DNA cells that are incorporated into the DNA, thereby "increasing the benefits of the gene editing process and possibly even more dramatic results."
"Specifically, we will examine the details of cell systems involved in DNA repair to refine the SATI technology even further for better DNA correction," said Reyna Hernandez-Benitez, co-author of the study.
Although, according to a study, it is reasonably controversial that it can cause genetic damage, CRISPR has been attributed several biological and biological damages to scientific achievements. This includes the elimination of viruses in pigs that could be harmful to humans and could be the basis for porcine organ transplantation to humans.
The gene editing tool also corrected a disease-causing gene mutation in human embryos and prevented the mutation from transitioning to future generations.
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