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A CRISPR clinical trial is currently underway at the University of Pennsylvania, where researchers are testing the effectiveness of gene modification technologies in the treatment of cancer patients.
"To date, two patients with recurrent cancers – multiple myeloma and sarcoma – have been treated in this trial," Penn Medicine's spokesman wrote in an e-mail sent to IFLScience. "The results of this study will be shared at a convenient time through a medical meeting presentation or a peer-reviewed publication."
CRISPR works by targeting certain genes responsible for certain functions or traits. Once found, an enzyme called Cas9 binds to the DNA, "cuts" it and closes that targeted gene. As early as last fall, the team began the process of removing cells from patients' immune systems and modifying them in the laboratory before reintroducing them into patients. Hopefully these modified cells will target and destroy the cancer cells, NPR reported for the first time. No information is available on the efficacy of the procedure in both patients, but researchers plan to treat 18 patients with various forms of cancer by the end of the trial in 2033.
If successful, the trial is one of many trials that could revolutionize the way diseases are prevented and treated.
Earlier this year, the use of CRISPR was causing concern after the Chinese researcher He Jiankui had used this technology to modify the binoculars' DNA, in order to give them resistance to the immunodeficiency virus human (HIV) before birth. moratorium of the research community on such practices. This controversial research differs radically from the clinical trial involving cancer patients in that it was conducted on human embryos and modified the inherited traits that could be transmitted for future generations. UPenn researchers provide medical treatment that only changes the DNA of participating individuals and, to their knowledge, would not carry the risk of transmitting modified genes to their children.
As noted by NPR, other human trials involving CRISPR are about to begin in the US, Canada, and Europe to test its effectiveness at treating various diseases, including two other CRISPR studies. based in the United States designed to treat genetic disorders of the blood. sickle cell disease and beta-thalassemia.
[H/T: NPR]
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