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Gene Editing Tool Hailed As A Breakthrough, And It Really Is One

Dec 28, 2015
Originally published on December 29, 2015 2:36 pm

Every once in a while a technology comes along that completely alters the way scientists do their work.

It's hard to imagine astronomy without a telescope or high energy physics without an accelerator.

From here on in, it's going to be impossible to imagine biology without CRISPR-Cas9.

Simply put, CRISPR-Cas9 allows scientists to make specific changes to specific genes in living cells. Such a thing was possible in the past using technques called zinc finger nucleases and Talens. But those techniques were cumbersome and weren't widely adopted.

In the three short years since the first scientific papers appeared about CRISPR-Cas9, the technique has been "spreading like wildfire," says Ramesh Akkina, a molecular immunologist at Colorado State University.

And for 2015, the journal Science called CRISPR the "breakthrough of the year."

There have been lots of ethical debates about what the new gene editing technique could do, such as creating designer babies or making mutant species. But most biologists aren't interested in making designer babies or mutant species. They just want to understand basic things like how the cells in our bodies work, or how certain genes function. They expect CRISPR-Cas 9 to be very, very helpful with those lines of inquiry.

Recently I visited Colorado State University to give a talk, but I realized it would be a great place to assess just how far and wide CRISPR-Cas9 had spread.

First, I talked with Christopher Allen, a scientist in the environmental and radiological health sciences department who studies the genes that are important for repairing DNA inside our cells.

When that repair process goes wrong, the result can be cancer. So Allen would like to be able to compare cells side by side: one that has a gene he thinks is important in the repair process, and one that is missing that gene. To do that, he has to modify the genome of a cell, something CRISPR-Cas9 will let him do easily.

Another scientist, Carol Wilusz, studies how and when genes are expressed in stem cells. She says CRISPR-Cas9 is going to make a difference in her work, "because it's going to enable us to do experiments that we've been struggling to do through other approaches."

Now, Wilusz and Allen aren't trying to cure diseases, at least not directly, but CRISPR might be useful for that, too.

"The work we're doing now is to use CRISPR-Cas technology to delete HIV genome from infected cells, such that the cell can be cured completely," says Ramesh Akkina, a virologist at Colorado State. Right now he is perfecting that trick in cells in the lab, but he is working on a scheme to do it in patients as well.

CRISPR-Cas9 isn't just useful for working organisms in the animal kingdom. It's useful in forests and fields.

"I study diseases of plants," says plant geneticist Jan Leach, "and my goal is to try to make plants that are resistant to different pathogens."

She says there are a host of genes inside plant cells that turn on when the plant tries to fight invasion from bacteria or fungi. She'd like to be able modify all of them. With CRISPR-Cas9, she can.

"I've been working in this particular area for over 20 years, and in 20 years I've been able to do one or two genes," says Allen. "With CRISPR-Cas I'll be able to do 20 or 30."

In two days, I spoke with nine different researchers. I asked them all the same question. "Will CRISPR-Cas9 have an impact on your work?"

Honestly, it's stunning to witness the impact CRISPR-Cas9 has had on biology. It ranks with the most important tools invented in the past century. I wouldn't be surprised if it wins Science magazine's breakthrough of the year next year too.

Copyright 2016 NPR. To see more, visit http://www.npr.org/.

Transcript

AUDIE CORNISH, HOST:

This year, a new invention has transformed biological research. It's called CRISPR-Cas9, and it allows biologists to manipulate genes in a way they've never been able to before. Science magazine calls CRISPR the breakthrough of the year. NPR science correspondent Joe Palca has been assessing the impact of new technologies as part of his series Joe's Big Idea. Earlier this month, he visited Colorado State University and has this report on why scientists are ga-ga about CRISPR.

JOE PALCA, BYLINE: The main reason I came to Colorado State was to give a talk about what I do covering science. But I realized that Colorado State would be a great example of how CRISPR-Cas9 has just changed the lives of scientists in this country. My first interview was in a building called MRB. I wasn't sure what MRB stood for until I read the sign out front.

Molecular and radiological biosciences - oh, MRB. I get it.

There's been lots of speculation about what the new gene editing technique could do - creating designer babies or making mutant species. But most biologists aren't interested in making designer babies or mutant species. They just want to understand basic things, like how the cells in our bodies work or how certain genes work, and they expect CRISPR-Cas9 to be very, very helpful with that.

CHRISTOPHER ALLEN: Hi there.

PALCA: Hi. I'm Joe. Nice to meet you.

ALLEN: Nice to meet you.

PALCA: I'm in the office of Christopher Allen.

ALLEN: I'm an assistant professor at Colorado State University in the environmental and radiological health sciences department.

PALCA: And what kinds of work do you do?

ALLEN: Cancer biology, focus on DNA repair.

PALCA: CRISPR is a big deal to this guy. Allen is studying which genes are important for repairing DNA inside our cells. When that repair process goes wrong, the result can be cancer. So Allen would like to be able to compare cells side-by-side - one that has a gene he thinks is important in the repair process and one that's missing that gene. To do that, he has to modify the genome of a cell.

ALLEN: There have been techniques that have been somewhat effective. But this CRISPR thing came along, and all of a sudden, it's a simple transfection of these plasmas that then will go in and do some work...

PALCA: OK. We veered into the technical stuff. But the key point here is CRISPR-Cas9 lets him make the comparisons he hopes will reveal more about the DNA repair process. So that's DNA repair.

Heading up to room 407, the office of Carol Wilusz.

She studies special cells called stem cells.

CAROL WILUSZ: I'm a professor of microbiology, immunology and pathology at Colorado State University.

PALCA: All three?

WILUSZ: Yes. It's a very big department.

PALCA: I guess so. Anyway, she's interested in which genes are turned on and off in stem cells, a process called gene expression. She says CRISPR-Cas9 is going to make a big difference in her work.

WILUSZ: Because it's going to enable us to do experiments that we've been struggling to do through other approaches.

PALCA: She says pretty much every kid on the block or down the hall that studies gene expression is going to be using CRISPR-Cas9 in their research. Now, Wilusz and Chris Allen aren't trying to cure diseases, at least not directly, but CRISPR might be useful for that, too.

I'm in the pathology building - sounds a bit like I've entered a sarcophagus. Ramesh Akkina's office is here. He's working with HIV, the virus that causes AIDS.

RAMESH AKKINA: The work we are doing is to use CRISPR-Cas technology to delete HIV genome from an infected cell such that the cell can be cured completely.

PALCA: Cured completely. Right now he's perfecting that trick in cells in the lab, but he's working on a scheme to do it in patients as well. Akkina says CRISPR-Cas9 is spreading through the science world like, well...

AKKINA: Almost like a wildfire (laughter). Everybody's really interested in this technology because it is so easy to do. And also, it's got such great potential in many, many, many areas not just in HIV.

PALCA: Even, dare I say it, botany.

Next appointment - plant sciences. I think it's kind of funny that plant sciences is just across the quad from animal sciences.

I'm here to talk to Jan Leach.

JAN LEACH: I study diseases of plants, and my goal is to try to make plants that are resistant to different pathogens.

PALCA: She says there are a host of genes inside plant cells that turn on when the plant tries to fight invasion from a bacteria or a fungus. She'd like to be able to modify all of them. With CRISPR-Cas9, she can.

LEACH: I've been working in this particular area for over 20 years, and in 20 years, I've been able to do one or two genes. With CRISPR-Cas, I'll be able to do 20 or 30.

PALCA: Twenty or 30 in just a few years or less. Like all the people I spoke with, Leach says she expects many of her colleagues will be using CRISPR-Cas9 in their research if they aren't already. In addition to the four scientists you just heard from, I spoke to five others. I asked them all if CRISPR-Cas9 will have an impact on their work.

GREGORY AMBERG: Yes, absolutely.

HEATHER SZERLONG: Absolutely.

JOSEPH RUSSO: Yes, big difference.

SANTIAGO DIPIETRO: Definitely.

GREG EBEL: Well, I hope so.

PALCA: That was Gregory Amberg, Heather Szerlong, Joseph Russo, Santiago DiPietro and Greg Ebel. Ebel hasn't started using CRISPR-Cas9 yet, but he plans to. I've been reporting on science now for more than three decades, and I have to say it's stunning to witness the impact CRISPR-Cas9 is having on research. I wouldn't be surprised if it wins breakthrough of the year again next year. Joe Palca, NPR News. Transcript provided by NPR, Copyright NPR.