DNA-editing studies could help prevent thousands of genetic diseases

Studies at MIT and Harvard could mark an end to cystic fibrosis and inherited blindness
Breakthrough: new DNA and RNA research could treat thousands of diseases
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Ben Travis27 October 2017

Scientific studies have demonstrated advances in the ability to ‘edit’ genetic code – a process which could eventually treat thousands of diseases and conditions.

Two studies, both undertaken at the Broad Institute of MIT and Harvard, showed off the ability to re-write DNA and edit RNA, a genetic material related to DNA.

The breakthrough could eventually help to prevent genetic diseases and mutations, and provide a ‘cure’ for the likes of cystic fibrosis and genetically-inherited blindness.

Tools called base editors, which can alter the building blocks of DNA, are key to the studies.

"We are hard at work trying to translate base editing technology into human therapeutics,” says Broad Institute professor David Liu, adding that there’s a way to go yet before the technology will have real-world application.

“Having a machine that can make the change you want to make is only the start. You still need to do all this other work, but having the machine really helps.”

Potentially thousands of diseases caused by single ‘typos’ in the genetic code could eventually be treated with the technology.

One study showed the ability to edit RNA in order change inherited anaemia in human cells.

"The ability to correct disease-causing mutations is one of the primary goals of genome editing,” said the Broad Institute’s Feng Zhang. “So far, we’ve gotten very good at inactivating genes, but actually recovering lost protein function is much more challenging. This new ability to edit RNA opens up more potential opportunities to recover that function and treat many diseases, in almost any kind of cell.”

The DNA study was published in scientific journal Nature, and the RNA study can be found in Science.

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