For the first time, doctors in the United States have used cutting-edge CRISPR technology to treat cancer patients. Two cancer patients participating in a study with the University of Pennsylvania in Philadelphia are being treated for myeloma and sarcoma with the technique.
First reported by NPR, the CRISPR study comes after the technique achieved a level of infamy around the world for being mishandled by Chinese scientist He Jiankui. After attempting to use the CRISPR techniques of genetic editing on infants, He faced the brunt of near-global condemnation from both his fellow scientists and the Chinese government.
But around the world, CRISPR is getting a welcome in much more traditional scientific operations than He's. There's a lot of hype around CRISPR, also known as CRISPR-Cas9 in recognition of the enzyme that uses CRISPR to recognize specific strands of DNA. In addition to cancer, scientists say that the technique "has shown promise in certain neurological disorders," including Huntington's Disease.
The issues with He's work were numerous, ranging from sloppy science to consent forms that didn't fully inform parents what was going to be done to their children. While He worked with American scientists from Stanford and Rice, the work was done outside of any institutional setting. He released his finding not in an academic journal, but on YouTube. There's hope that, while CRISPR remains an experimental technique, within an institutional framework the sort of basic problems that plagued He's work will not replicate themselves.
"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 speaking to NPR.
Studies looking at how CRISPR could affect conditions ranging from sickle cell to inherited blindness could be up and running soon. The blindness study, sponsored by Editas Medicine, Inc., of Cambridge, Massachusetts, will attempt to edit genes within the human body.
The study from the University of Pennsylvania, run by Dr. Edward Stadtmauer, will remove immune system cells from patients. Then, scientists will attempt to genetically alter them in a lab. Afterwards, the cells will be reintroduced to each patient's body. If all goes according to plan, the cells will target and eliminate the cancerous cells. The study features 18 patients.
So far, that's all Penn is saying."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 to NPR. That's already a big step up from He Jiankui's work.
Now that it appears to be clearing the standards of scientific ethics, the world will be watching closely.
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