"Sickle-Cell Anaemia" by Osaretin / CC BY-SA 4.0

Quest to use CRISPR against disease gains ground

Jan 8, 2020

By Heidi Ledford

The prospect of using the popular genome-editing tool CRISPR to treat a host of diseases in people is moving closer to reality.

Medical applications of CRISPR–Cas9 had a banner year in 2019. The first results trickled in from trials testing the tool in people, and more trials launched. In the coming years, researchers are looking ahead to more sophisticated applications of CRISPR genome editing that could lay the foundation for treating an array of diseases, from blood disorders to hereditary blindness.

But although the results of clinical trials of CRISPR genome editing so far have been promising, researchers say that it is still too soon to know whether the technique will be safe or effective in the clinic.

“There’s been a lot of appropriate caution in applying this to treating people,” says Edward Stadtmauer, an oncologist at the University of Pennsylvania in Philadelphia. “But I think we’re starting to see some of the results of that work.”

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