DNA snipping technique could transform gene therapy

BERKELEY 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.

"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."

"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)."

The latter technique made it easy to generate millions of copies of small pieces of DNA and permanently altered biological research and medical genetics.

Two developments - zinc-finger nucleases and TALEN (Transcription Activator-Like Effector Nucleases) proteins have gotten a lot of attention recently, including being together named one of the top 10 scientific breakthroughs of 2012 by Science magazine. The magazine labeled them "cruise missiles" because both techniques allow researchers to home in on a particular part of a genome and snip the double-stranded DNA there and there only.

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DNA snipping technique could transform gene therapy