medical science to cure genetic disorders?
Is it possible that medical science will one day be able to cure genetic disorders?
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According to recently published research, the treatment of debilitating inherited diseases is one of the most significant challenges confronted by contemporary medicine. Patients and their families have been given new hope as a result of the introduction of CRISPR technology and the advancements that have been made in genetics research over the past ten years. Despite this, there is still a serious concern regarding the safety of these novel techniques.
A group of biologists from the University of California, San Diego, including postdoctoral researcher Sitara Roy, expert Annabel Guichard, and professor Ethan Bier, have described a novel method that is safer than previous techniques for fixing genetic flaws. This method may one day be used. Their method, which makes use of the body's built-in DNA repair mechanisms, lays the groundwork for cutting-edge gene therapy approaches that have the potential to treat a wide range of genetic disorders. Additionally, their method utilises the body's built-in DNA repair mechanisms.
According to Guichard, the senior author of the study, "the healthy variant can be used by the cell's repair machinery to correct the defective mutation after cutting the mutant DNA." "Remarkably, this can be achieved even more efficiently by a simple harmless nick," he added.
Researchers working with fruit flies developed mutants that allowed for the visualisation of a process known as "homologous chromosome-templated repair," or HTR. This was accomplished by observing the production of pigments in the flies' eyes. These mutants had eyes that were completely white when they were first created. However, when the same flies expressed CRISPR components (a guide RNA in addition to Cas9), they displayed large red patches across their eyes. This was a sign that the cell's DNA repair machinery had been successful in reversing the mutation by making use of the functional DNA from the other chromosome.
After that, they put their newly developed system to the test by using variants of Cas9 called "nickases"
that targeted only one strand of DNA rather than both of them. Surprisingly, the authors discovered that such nicks also resulted in a high-level restoration of the fly's natural red eye colour, almost reaching the level of normal, healthy flies that have not been mutated. They discovered that the nickase had a repair success rate of between 50 and 70 percent, whereas the dual-strand cutting Cas9 only had a success rate of between 20 and 30 percent. Dual-strand cutting Cas9 also generates frequent mutations and targets other sites throughout the genome (so-called off-target mutations). Roy, who was the primary author of the study, stated that he "could not believe how well the nickase worked; it was completely unexpected." [Citation needed] The researchers pointed out that due to the new system's adaptability, it might be possible to use it as a template for correcting genetic mutations in mammals.
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