REVOLUTIONARY GENE-EDITING TOOL MIGHT BE THE MOST CONSEQUENTIAL DISCOVERY SO FAR THIS CENTURY, SAYS A LEADER OF THE HUMAN GENOME PROJECT ON "60 MINUTES"

Hope Is to Use Gene-Editing Tool CRISPR to Treat Thousands of Diseases

Can a discovery that has already restored sight in genetically blind mice do the same in humans? Could it one day be used to edit out hereditary illnesses like heart disease from an embryo? These are very real possibilities thanks to CRISPR. A leader of the Human Genome Project believes this gene-editing tool could be the most consequential discovery in biomedicine this century. Bill Whitaker reports on a new tool that's revolutionizing biomedical research on the next edition of 60 MINUTES Sunday, April 29 (7:00-8:00 PM, ET/PT) on the CBS Television Network.

Eric Lander, director of the Broad Institute in Cambridge, Mass., was a leader in the project to map all the genes in the human body; without that body of knowledge, CRISPR would not be possible. "During the Human Genome Project, we could read out all the human DNA, and then, in the years afterwards, find the misspellings that caused human diseases," he tells Whitaker. "But we had no way to think about how to fix them. And then, pretty much on schedule, this mind-blowing discovery that bacteria have a way to fix those misspellings appears."

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. It was discovered by examining the way bacteria defends itself against invading viruses. Bacteria keep a piece of that DNA as a reminder, and if the same virus invades again, bacteria can identify it and disable it. Feng Zhang has worked at the Broad Institute for seven years and figured out how to make CRISPR work in human cells. Just as bacteria are able to track down a virus, CRISPR can be programmed to locate faulty DNA and edit it, adding in synthetic DNA. "You can give the cell a new piece of DNA that carries the sequence you want to incorporate into the genome," says Zhang.

The possibilities of this process are wide and deep. It can be used on practically any living thing. The technique has already created malaria-resistant mosquitoes and modified rice to better withstand droughts - just two of the countless applications. CRISPR is readily available to medical researchers, some of whom have attacked diseases in animals successfully. Zhang says the CRISPR from his lab has been sent out over 45,000 times to 2,200 labs in 61 countries. "There are about six thousand or more diseases that are caused by faulty genes. The hope is that we will be able to address most, if not all of them."

Lander says the implications of what could be been done with CRISPR in embryos - editing out disease or editing in desirable traits before birth - should not be the emphasis now. The focus should be on people with disease. "I don't think we're close to ready to use it to go edit the human population. I think we've got to use it for medicine for a while. I think those are the urgent questions. That's what people want right now... cures for disease."

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