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TREATMENT OF GENETIC DISEASES MAY NOW BE POSSIBLE!
A new method, described as a turning point, can correct as many as 75 thousand harmful gene changes. According to the first trials, scientists performed 175 successful gene changes.
Scientists who invented a powerful new molecular tool that corrects the vast majority of mutations that cause genetic diseases in humans have created hope for the treatment of genetic disorders, according to information from BBC.
The new procedure has the ability to correct about 89 percent of the 75 thousand or so harmful gene mutations that lead to many diseases such as cystic fibrosis, sickle cell anemia, and which destroy nerves.
The study, described as a turning point, opens the door to a new era of genome editing. But the researchers note that more research is needed before the procedure can be safely used in humans.
Another obstacle is ensuring that the molecular tool can be sent in sufficient quantities to the cells involved to treat a disorder.
Important study from Turkish scientists: genetic causes of heart rhythm disorder were determined important study from Turkish scientists: genetic causes of heart rhythm disorder were determined
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HOW IS THE GENE REGULATED?
In the new method developed by scientists, the main regulator contains a protein designed to guide it to its target. The regulator uses an enzyme called cas9 to open the relevant DNA chain when it reaches the target, producing new DNA that will be added to the region in the second step.
Another guiding protein sends the main regulator to the as-yet unregulated opposite side of the DNA chain. The chain of cells re-repairs the edited DNA after copying and inserting it.
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175 ATTEMPTS WERE SUCCESSFUL
The ability to rewrite the genetic code is one of the most striking scientific developments in recent years. Instead of the method that divides the DNA chain in half, the procedure writes a new DNA to the corresponding sequence by “creating a notch” the chain.
The paper, published in the journal Nature, detailed how they performed 175 effective gene changes in human cells using the new method.
Before the new method was developed, it was not possible to make such a variety of DNA changes in different types of cells without adding or releasing other genetic residues that could interfere with the functioning of the regulated cell.
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