The new technology implementing gene modifying may be very helpful, determining treatments for genetic blood diseases. Some scientists from Yale University and Carnegie Mellon University have teamed up to analyze and test the gene-modifying system. They used as subjects mice, and they examined the effects of their new technology. The study was published in the Nature Communications magazine.
In the research, scientists have used CRISPR which represent clustered regularly interspaced short palindromic repeats. Whereas other conventional methods have conducted the research by relying on DNA enzymes to identify particular genes, this team avoided this practice. The author of the study claimed that their contribution might be categorized as a revolutionary breakthrough in the field of medicine.
The experts’ team developed a system based on the nanoparticles approved by FDA meant to deliver the PNA molecule together with part of a DNA. They were prone to repair an impaired gene in the mice’s DNA. Danith Ly, one of the scientists conducting the study, has argued that this procedure has not been accomplished with CRISPR.
Besides the tests carried out on mice, researchers have tried to apply their theory to human bone cells. Analyzing this in their lab, the team observed an increased rate of success for the gene-modifying technology. They registered up to 7% increase in the positive results they were expecting to appear. Specialists have also revealed high levels of hemoglobin a few months after the treatment.
Despite this insignificant percentage for some, this could develop to become a revolutionary project in time. The study could evolve in such a way that it could register significant outcome. If there appeared a 7% chance of success, it means that these chances could grow depending on the work of scientists.
The problem with this DNA-cutting technology is twofold. Firstly, the enzymes are too broad to be adequately distributed to living animals in a direct way. Thus, researchers need to remove those cells, underwent treatment for them, and then they placed them into the body. Secondly, once the experts were analyzing the enzymes located inside the cell, those enzymes could haphazardly cut unnecessary part of the DNA that was not targeted to be examined. The precision of this procedure was crucial. The Carnegie Mellon-Yale system prevented using one of these two possibilities.
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