CRISPR Successfully Treats Genetic Disorder in Adult Mammal

First Posted: Jan 01, 2016 01:19 PM EST

CRISPR, the gene editing tool, has long been touted as something that could change the way we treat genetic disorders. Now, scientists have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy.

In this latest study, the researchers used CRISPR to correct genetic mutations in cultured cells from Duchenne patients, and other labs had corrected genes in single-cell embryos in a laboratory environment. But the latter approach is currently unethical to attempt in humans, and the former faces many obstacles in delivering treated cells back to muscle tissues.

Another approach, which involves taking CRISPR directly to the affected tissues through gene therapy techniques, also faces challenges, particularly with delivery.

In this latest study, the researchers overcame several of these obstacles by using a non-pathogenic carrier called adeno-associated virus, or AAV, to deliver the gene-editing system.

"Recent discussion about using CRISPR to correct genetic mutations in human embryos has rightfully generated considerable concern regarding the ethical implications of such an approach," said Charles Gersbach, one of the researchers, in a news release. "But using CRISPR to correct genetic mutations in the affected tissues of sick patients is not under debate. These studies show a path where that's possible, but there's still a considerable amount of work to do."

The findings could be huge when it comes to using these therapies in the future. With that said, it will take quite a bit more research on gene editing before these types of treatments can be used on humans.

The findings are published in the journal Science.

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