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Muscular dystrophy could be the next disease to get whacked by gene therapy

Four boys have received a gene-replacement treatment for muscular dystrophy, and it’s stoking hopes that scientists will cure this disease for good.

What: The biotech company Sarepta, in Cambridge, Massachusetts, said boys who received the gene therapy showed high levels of dystrophin, the protein that’s missing in the terrible disease.

Big deal? You bet. Muscular dystrophy is common, fatal, and currently incurable. But gene therapy might solve it. Sarepta’s stock shot up today by 50 percent.

How they did it: Dystrophin is one of the biggest genes in the human genome. Its size makes it too big to stuff inside a virus, which is needed to get the new gene into the body. The Sarepta team got over this hurdle by working with foreshortened “micro” versions of the gene instead. Others are eyeing CRISPR gene editing as the way to go.

More to come: Sarepta’s study is being carried out at Nationwide Children’s Hospital in Ohio. Two other similar studies, from Pfizer and Solid Biosciences, are also under way.

Deep Dive


These scientists used CRISPR to put an alligator gene into catfish

The resulting fish appear to be more resistant to disease and could improve commercial production—should they ever be approved.

Next up for CRISPR: Gene editing for the masses?

Last year, Verve Therapeutics started the first human trial of a CRISPR treatment that could benefit most people—a signal that gene editing may be ready to go mainstream.

CRISPR for high cholesterol: 10 Breakthrough Technologies 2023

New forms of the gene-editing tool could enable treatments for common diseases.

An ALS patient set a record for communicating via a brain implant: 62 words per minute

Brain interfaces could let paralyzed people speak at almost normal speeds.

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Illustration by Rose Wong

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