Feng Zhang is one of the founders of Editas Medicine, which aims to use CRISPR gene-editing technology to treat disease. Image: Kent Dayton

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Instead of taking prescription pills to treat their ailments, patients may one day opt for genetic 'surgery' using an innovative gene-editing technology to snip out harmful mutations and swap in healthy DNA.

The system, called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), has exploded in popularity in the past year, with genetic engineers, neuroscientists and even plant biologists viewing it as a highly efficient and precise research tool. Now, the gene-editing system has spun out a biotechnology company that is attracting attention from investors as well.

Editas Medicine, based in Cambridge, Massachusetts, announced its launch on 25 November with an initial $43 million venture capital investment. The company, founded by five leading CRISPR researchers, aims to develop therapies that directly modify disease-related genes.

"This is a platform that could have a profound impact on a variety of genetic disorders," says interim president Kevin Bitterman, a venture capitalist at Polaris Partners in Waltham, Massachusetts, which is one of Editas' backers.

The nicest cut CRISPR piggybacks on an immune strategy that bacteria use to detect and chop up foreign DNA. The DNA-cutting enzyme Cas9 finds its target with the help of an RNA guide sequence that researchers can now engineer to home in on potentially any gene of interest.

Editas is not disclosing its intended targets, but the technology might be tried first on diseases caused by a single faulty gene copy, says Feng Zhang, a neuroscientist at the Massachusetts Institute of Technologys McGovern Institute for Brain Research in Cambridge, Massachusetts, and one of Editas founders. Simply disabling the disease-causing copy could clear the way for the good copy to take over. Treating conditions involving two dysfunctional gene copies will require correcting the gene by splicing in healthy DNA a feat that Zhang says will require more work and engineering.

See the article here:
'Genetic Surgery' May Be Enabled by a New Technology ...

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