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And More about CRISPR/Cas9

Genome editing, particularly in the form of the CRISPR/Cas9 approach, offers both opportunities and risks, writes Jennifer Doudna from University of California, Berkeley in Technology Review.

Doudna and her colleague Emmanuelle Charpentier at UmeåUniversityreported on the approach in Science in 2012, while the Broad Institute's Feng Zhang patented the approach after showing he invented it first. (TheUniversity ofCalifornia has asked the patent office to review that decision.)

The CRISPR/Cas9 system, Doudna says, is "simple and effective" and has so far been used develop new crop strains, cure a genetic liver disease in mice, and enable biofuel synthesis in fungal cells. "The CRISPR-Cas9 technology has opened up a world of research opportunities that were inconceivable just three years ago," she adds.

However, since the approach could be used to alter germ cells or embryos, Doudna notes that there are risks involved.

Indeed, researchers from Sangamo BioSciences, the Alliancefor Regenerative Medicine, and elsewhere called for a moratorium on any such germline editing. In their Nature commentary, they warned that alterations to the germline with current tools "could have unpredictable effects on future generations."

Doudna says that further research into the "utility and risks of CRISPR-Cas9 in cells including human germ cells, as well as the risks inherent in any human clinical applications that might be possible in the future" is needed. She adds that while researchers "should embrace this technology, scientists also must come together to guide peers and regulators as to its responsible use."

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