For the first time in the USA
Representatives of the University of Pennsylvania confirmed the first use of CRISPR technology in humans in the United States
Dmitry Mazalev, Naked Science
A promising gene editing technology called CRISPR has been in the news a lot lately. So, last year, Chinese scientist He Jiankui stunned the world with his admission that he used CRISPR technology to create genetically modified children. At the same time, scientists hope that this technique will allow for very precise DNA modifications, which will eventually help in the treatment of many diseases. Finally, scientists have begun to take the first steps to make this dream a reality.
In June 2016, the US National Institutes of Health (National Institutes of Health) provided the University of Pennsylvania (UPenn) research group with the opportunity to begin testing CRISPR technology in humans, but only now has a representative of UPenn confirmed that researchers have officially started using it. The use of CRISPR in humans will be the first national event that could lead to more widespread use of the technology in the future.
A university spokesperson told NPR that the UPenn team has already used CRISPR technology to treat two cancer patients: one patient with multiple myeloma and one with sarcoma. Both relapsed after standard treatment. In the course of the work, the researchers removed immune system cells from patients, using CRISPR to edit cells (target them to tumors), and then returned them to the patients' organisms. According to a representative of UPenn, the results of the study will soon be presented at one of the medical congresses or published in the relevant journal.
Traditional gene therapy uses viruses to inject new genes into cells to treat diseases. CRISPR technology largely avoids the use of viruses, which cannot but cause some security concerns. Instead, scientists directly make changes to DNA using targeted molecular tools. The technique can be compared to the cut–and-paste function in a word processing program - it allows scientists to remove or modify certain genes that cause the problem.
The work of scientists from Pennsylvania may be the first application of CRISPR technology on humans in the United States, but other researchers are also working on this method. According to one of the experts, 2019 may be a key year for determining what CRISPR technology can do useful for the world and how it will help in the treatment of a variety of diseases.
According to experts UPenn, there is an important difference between their medical research and what the Chinese scientist He Jiankui did. He used CRISPR to edit the genes of human embryos: this means that the changes he made will be passed on to future generations. And he did it before most scientists decided that it was safe, amid calls to introduce a moratorium on editing genes of hereditary traits. As for medical treatment, modifications are made only to the DNA of individual patients. Thus, such gene editing does not cause dystopian concerns about the reorganization of the human race.
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