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New gene editing technology can correct 89% of genetic defects



The new technique is called "main editing," and was developed by researchers at the MIT and Harvard Broad Institute, who published their findings Monday in the journal Nature.

Basic editing is based on the powerful editing of CRISPR genes, but is more precise and multifunctional – it "directly writes new genetic information to a specific DNA site," according to the document.

In the traditional approach CRISPR-Cas9 Cas9, a type of modified protein, acts as a pair of scissors that can snap parts of strands of DNA. It can target genes at a specific location – for example, to disrupt a mutation.

About two-thirds of the known genetic variants of humans associated with the disease are genetic mutations at one point, so that genetic editing has the potential to correct or reproduce such mutations.

  Scientists edit gene for blood disease in human embryos

Industrial editing CRIS9 editing CRIS9 , which can generate new DNA. The tool cuts the strand of DNA and then transfers the edited sequence to the target DNA – allowing researchers to seamlessly insert and delete parts of human cells.

The technique allows researchers to search for and replace entire stretches of DNA strands, all without destructive breaks or donor DNA. With this method, researchers say they hope to correct up to 89% of the known genetic variants causing the disease accurately and efficiently.

"With initial editing, we can directly correct the sickle cell anemia mutation back to normal sequence and remove the four additional DNA bases that cause Tay Sachs disease without cutting the DNA completely or needing DNA templates." says David Liu, one of the authors of the study, in a press release from the Broad Institute.
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"The versatility of original editing is quickly becoming apparent as we develop this technology," says Andrew Anzalone, another author of the study, in a statement on the press. "The fact that we could directly copy new genetic information to a target site was a revelation. We were really excited."

The team of researchers will continue to work to refine the technique, trying to maximize its effectiveness in different cell types. and investigation of possible effects on cells. They will also continue to test different disease models to ultimately "provide a potential avenue for therapeutic applications in humans," according to a press release.

Gene editing is still a relatively young and rapidly expanding field of study – CRISPR-Cas9 is based on the discovery of a decade, but first used only in humans in 2016. Then in 2017, the Broad Institute developed a new technique called base editing that can make changes to the target DNA site without cutting the DNA.
  The proposal for a global moratorium on editing DNA is met with criticism

Institute, and elsewhere, we hope CRISPR will one day target a wide range of "bad" genes – potentially helping people avoid obesity, Alzheimer's disease, genetic forms of deafness, and more.

However, with the advancement of technology, doctors, scientists, and bioethicists have also raised ethical questions. Some fear that it may open the door for human embryos to be manipulated for non-therapeutic reasons, or that it may create unwanted mutations and new diseases.

Shortly earlier this year, in March, a group of researchers, including a scientist, who created and patented CRISPR technology called for a global moratorium on human germline editing – changes made to inherited DNA that could be transmitted to the next generation.

They listed ethical issues and cited Chinese scientist He Junqoui, who claimed to have made gene editorials on the creation of two AIDS-resistant babies last year. His work, which could have unintended consequences, has been internationally condemned and called "disgusting by nature" by the Chinese authorities.

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