CRISPR Improves Disease in Adult Mice

Three groups of researchers used the gene-editing method to restore a protein deficient in Duchenne muscular dystrophy.

kerry grens
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WIKIMEDIA, CENTERS FOR DISEASE CONTROL AND PREVENTION'S PUBLIC HEALTH IMAGE LIBRARYCRISPR has fixed the protein problems in adult mice that lie at the root of Duchenne muscular dystrophy (DMD), a progressive disease that saps kids of muscle strength and ultimately shortens their lives. Scientists had succeeded in using the gene-editing technique to restore protein function in human cells or mouse embryos, but this is the first time adult animals have been treated.

“The hope for gene editing is that if we do this right, we will only need to do one treatment,” Duke University’s Charlie Gersbach, who led one of three independent research teams that published results in Science last week (December 31), told The New York Times. “This method, if proven safe, could be applied to patients in the foreseeable future.”

The problematic protein is called dystrophin. All three groups took the same approach, first demonstrated in mouse embryos by Eric Olson of the University of Texas Southwestern Medical Center in 2014, to correct dystrophin deficiencies. They clipped a mutant exon from the gene for dystrophin, resulting in a truncated but functional protein. “Importantly, in principle, the same strategy can be applied to numerous types of ...

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Meet the Author

  • kerry grens

    Kerry Grens

    Kerry served as The Scientist’s news director until 2021. Before joining The Scientist in 2013, she was a stringer for Reuters Health, the senior health and science reporter at WHYY in Philadelphia, and the health and science reporter at New Hampshire Public Radio. Kerry got her start in journalism as a AAAS Mass Media fellow at KUNC in Colorado. She has a master’s in biological sciences from Stanford University and a biology degree from Loyola University Chicago.

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