January 08, 2013
New Gene Editing Technique To Revolutionize Gene Therapy?

Tired of waiting for the biotech revolution? It is getting closer. A bunch of scientists are enthusing about a new way to insert genes into human cells.

A simple, precise and inexpensive method for cutting DNA to insert genes into human cells could transform genetic medicine, making routine what now are expensive, complicated and rare procedures for replacing defective genes in order to fix genetic disease or even cure AIDS.

Discovered last year by Jennifer Doudna and Martin Jinek of the Howard Hughes Medical Institute and University of California, Berkeley, and Emmanuelle Charpentier of the Laboratory for Molecular Infection Medicine-Sweden, the technique was labeled a "tour de force" in a 2012 review in the journal Nature Biotechnology.

That review was based solely on the team's June 28, 2012, Science paper, in which the researchers described a new method of precisely targeting and cutting DNA in bacteria.

Out with the expensive, complicated, and rare procedures for replacing defective genes. Enough of the snails pace rate of progress. Time to go to town fixing and improving our DNA.

Two new papers published last week in the journal Science Express demonstrate that the technique also works in human cells. A paper by Doudna and her team reporting similarly successful results in human cells has been accepted for publication by the new open-access journal eLife.

A simple, precise and inexpensive method for cutting DNA to insert genes into human cells could transform genetic medicine, making routine what now are expensive, complicated and rare procedures for replacing defective genes in order to fix genetic disease or even cure AIDS.

Discovered last year by Jennifer Doudna and Martin Jinek of the Howard Hughes Medical Institute and University of California, Berkeley, and Emmanuelle Charpentier of the Laboratory for Molecular Infection Medicine-Sweden, the technique was labeled a "tour de force" in a 2012 review in the journal Nature Biotechnology.

That review was based solely on the team's June 28, 2012, Science paper, in which the researchers described a new method of precisely targeting and cutting DNA in bacteria.

Two new papers published last week in the journal Science Express demonstrate that the technique also works in human cells. A paper by Doudna and her team reporting similarly successful results in human cells has been accepted for publication by the new open-access journal eLife.

Existing techniques are too slow. Faster and easier techniques will speed up rates of iteration by experimenters and make it easer to create therapies.

"The ability to modify specific elements of an organism's genes has been essential to advance our understanding of biology, including human health," said Doudna, a professor of molecular and cell biology and of chemistry and a Howard Hughes Medical Institute Investigator at UC Berkeley. "However, the techniques for making these modifications in animals and humans have been a huge bottleneck in both research and the development of human therapeutics.

"This is going to remove a major bottleneck in the field, because it means that essentially anybody can use this kind of genome editing or reprogramming to introduce genetic changes into mammalian or, quite likely, other eukaryotic systems."

"I think this is going to be a real hit," said George Church, professor of genetics at Harvard Medical School and principal author of one of the Science Express papers. "There are going to be a lot of people practicing this method because it is easier and about 100 times more compact than other techniques."

Revolutionized genome engineering is in the offing.

"Based on the feedback we've received, it's possible that this technique will completely revolutionize genome engineering in animals and plants," said Doudna, who also holds an appointment at Lawrence Berkeley National Laboratory. "It's easy to program and could potentially be as powerful as the Polymerase Chain Reaction (PCR)."

I'm thinking the 2020s will be when the gene therapies and cell therapies make a huge impact on human health, performance, and longevity. Maybe the late 2010s. But for the next few years we aren't going to see much at the clinical level.

Share |      Randall Parker, 2013 January 08 08:06 PM 


Comments
David Yerle said at January 9, 2013 3:56 AM:

Are we talking single-cell modifications or across the body modifications? I'm thinking only the latter type would be truly useful, but for that you need some kind of agent (a virus or other). I clicked on the link but found no further information...

JP Straley said at January 9, 2013 7:32 AM:

Even if it is just for single cell organisms, think what it means to projects like extracting oil from algae or for that matter, alcohol from yeast. Just make sure all those yeasts can use 5-carbon sugars and --- whammo ! --- a big yield increase. There are many other targets.

As for multicellular organisms, it's sure to be more complex...but what an advance we see here!

Lono said at January 9, 2013 8:28 AM:

This is very very promising. The acceleration of these bio-technologies is happening exactly like Randall, Kurzweil, and many other visionaries have predicted. This technology will allow us to create very specific viruses which will then allow for across the body modification - where and when necessary.

Now we only have to overcome the political and social obstacles in the way of making these therapies available to a greater citizenry.

Abelard Lindsey said at January 9, 2013 10:01 AM:

The main hurtle is excessive FDA regulation.

Randall Parker said at January 9, 2013 8:50 PM:

David,

First off, do not underestimate the value of single cell alterations. Their great value: to create great starting cells for cell therapies. The way it will work: Isolate cells from various locations in your body. Grow up cell lines. Do whole genome sequencing on each of the lines. Identify what's wrong with each line (accumulated mutations from aging). Choose the best lines and for each of them apply genetic edits to fix them. Then grow up the lines more, sequence again, and choose which ones look like their edits had the most success. Then use those as starting cells to create assorted cell therapies for muscles, livers, stomachs, vasculature, etc.

Also, use those heavily genetically edited cells to make replacement organs.

As for whole body: It is hard to get 100% coverage or anywhere near it. Dosing will be too uneven. But if, say, your body doesn't make enough of a hormone just grow a new thyroid or adrenal gland, or parathyroid. Or create some new Isle of Langerhams cells for insulin production.

What I really want: genetic editing to create youthful immune cells. They can be improved to make them more anti-cancer than even young cells.

Brett said at January 10, 2013 1:07 PM:

And it looks like we will be getting two new Nobel laureates.

Brett said at January 10, 2013 1:09 PM:

two new female Nobel laureates, I meant to say.

Reilly said at January 11, 2013 10:41 AM:

This sounds great! I'm looking forward to where the technology will be by 2020.

James Bowery said at January 11, 2013 4:12 PM:

One of the greatest boons to the FDA is the fact that people who might become suicide bombers are too debilitated by the FDA to do so.

Nanonymous said at January 12, 2013 11:03 AM:

Yes, unfortunately, the whole genome sequencing of the stem cells before and after editing will be essential. I am pretty sure with some low frequency even the best sequence-targeted nucleases will cleave random DNA non-specifically. Because there are 10^9 more places where they should not be cutting, in the end for every successful edit there probably will be few spurious edits elsewhere in the genome.Thanks to the predominance of junk DNA, most of these will be benign though.

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