CRISPR Inches Toward the Clinic

The gene-editing technology is already in trials for some rare conditions, with more human testing on the horizon.

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Since its debut less than a decade ago, CRISPR-Cas9 gene editing has inspired its share of grandiose and cautionary forecasts: that we might soon be able to resurrect beasts from the ancient past, for example, or (that classic genetic manipulation controversy) create designer babies. While such applications may never see the light of day, the technology is already revolutionizing genetics research, allowing scientists to easily manipulate model organisms in the lab. Moreover, many biomedical scientists see the system as a means to fix problematic DNA at play in countless genetic diseases.

Scientists and companies at the front lines of developing CRISPR-based therapies have started with relatively modest goals, targeting rare single-gene disorders and largely aiming to transplant modified cells rather than set a gene-editing delivery system loose in the body. But if green-lighted by regulators, such therapies could serve as trial balloons for a much broader use of CRISPR in ...

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Meet the Author

  • Shawna Williams

    Shawna was an editor at The Scientist from 2017 through 2022. She holds a bachelor's degree in biochemistry from Colorado College and a graduate certificate and science communication from the University of California, Santa Cruz.

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