Clinical studies of CRISPR/Cas9 technology
In the USA, genetic experiments on humans are allowed
Experts of the Advisory Committee on Recombinant DNA (Recombinant DNA Advisory Committee, RAC) approved the application of the University of Pennsylvania to conduct tests on human genetic modification (so in the text – VM) using CRISPR/Cas9 technology, reports the journal Science (First proposed human test of CRISPR passes initial safety review). During the experiment, immune cells will be improved, which will effectively fight cancer.
Scientists are going to change several genes at once, which early methods of genome modification did not allow. Researchers will isolate immune system cells – T-lymphocytes – from cancer patients, which will then be injected with neutralized viruses. The latter serve as a kind of transport for the delivery of the gene encoding the NY-ESO-1 protein receptor. This protein is often present on the surface of cancer cells, and almost never on the membrane of healthy ones.
CRISPR will also disrupt the PD-1 gene, which has been shown to weaken the activity of lymphocytes. The modified lymphocytes then return to the patient's body, where they begin to actively attack the tumor.
The two-year studies will involve 18 people with various types of cancer: myeloma, sarcoma and melanoma. Tumors should be resistant to existing treatments. Three research centers are going to conduct experiments: the University of Pennsylvania, the University of California at San Francisco and the University of Houston.
Some of the members of the commission, considering the application, expressed concern that the University of Pennsylvania has financial interests in the successful conduct of the experiment, since it has patents for the use of modified T-lymphocytes for the treatment of cancer. According to the experts' decision, the university should precisely define its role in the study, whether it will simply supply immune cells or directly participate in the trials. In the end, the committee unanimously approved the experiment.
The Advisory Committee on Recombinant DNA is an organization under the US Department of Health that deals with approval, as well as recommendations for the development of methods for modifying or synthesizing DNA.
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23.06.2016