In The U.S., CRISPR Experiment Being Used To Treat Cancer Patients At Upenn

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CRISPR gene editing technology allows scientists to make highly precise modifications to DNA.

Scientists have long been hopeful that CRISPR, a gene-editing technique that allows scientists to make precise DNA modifications, could help cure some diseases. A CRISPR study at the University of Pennsylvania in Philadelphia is now taking a step in that direction. On Monday, a university spokesperson said that two patients were treated using CRISPR, according to NPR.

One of the treated patients had multiple myeloma and the other had sarcoma. Both patients relapsed after they went through the standard treatment.

Many other human trials of CRISPR are set to begin in the U.S., Canada, and Europe to test its ability to treat certain diseases.

"2019 is the year when the training wheels come off and the world gets to see what CRISPR can really do for the world in the most positive sense," says Fyodor Urnov, a gene-editing scientist at the Altius Institute for Biomedical Sciences in Seattle and the University of California, Berkeley.

Traditionally, gene therapy uses viruses to treat diseases by inserting new genes into cells. CRISPR avoids the use of viruses and instead makes direct changes to the DNA. this allows scientists to remove or change specific genes which are problematic.

The University of Pennsylvania study involves removing immune system cells from the cancerous patient, genetically modifying the cells in a lab, and then infusing the cells back into the patient’s body. It is hoped that the new cells would destroy cancer cells.

"Findings from this research study will be shared at an appropriate time via medical meeting presentation or peer-reviewed publication," a university spokesperson wrote in an email.

CRISPR is also being used to attempt to treat sickle cell disease and another blood disorder, beta thalassemia. Another study hopes to treat an inherited form of blindness with CRISPR. The condition is known as Leber congenital amaurosis.

Still, there is some concern that CRISPR could not work and instead cause unintended changes in DNA.

"Every human on the planet should hope that this technology works. But it might work. It might not. It's unknown," says Laurie Zoloth, a bioethicist at the University of Chicago. "This is an experiment. So you do need exquisite layers of care. And you need to really think in advance with a careful ethical review how you do this sort of work."

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