Main Category: Genetics
Also Included In: Stem Cell Research; Arthritis / Rheumatology
Article Date: 29 Jul 2013 – 0:00 PDT
Duke University
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Simple and versatile tool enables broad-scale genome tinkering
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Gersbach now hopes to apply the new tool along with collaborators in the IGSP to investigate the functions of thousands of sites across the genome. With tissue engineer Farshid Guilak, a professor of engineering and orthopaedic surgery, he will continue to work on its application in the fight against inflammatory and autoimmune diseases such as arthritis.
Then, he and post-doctoral researcher Pablo Perez-Pinera found out about an RNA-guided protein called Cas9 found in a Streptococcus bacteria. The bacteria rely on Cas9 as part of an adaptive immune system to defend themselves against infection by viruses, cutting out a piece of the viral DNA and inserting it into their own genome for recognition of future infection. Other scientists then showed that those immune system components could function inside human cells.
The work was funded by the National Institutes of Health, the National Science Foundation and the American Heart Association. Other collaborators on the study include D. Dewran Kocak, Christopher Vockley, Andrew Adler, Ami Kabadi, Lauren Polstein, Pratiksha Thakore, Katherine Glass, David Ousterout and Kam Leong.
“This simple and versatile tool makes it easy for anyone to do this,” Gersbach says.
The new method also has obvious utility for gene therapy and for efforts to reprogram stem or adult cells into other cell types – for example, to make new neurons from skin cells.
In other words, it works, and it works on genes that matter from a clinical perspective. In principle, the RNA-guided tool could be used to modify or influence any gene anywhere in the genome.
Gersbach’s team recognized the RNA-guided nature of this system as a potential game-changer for the gene engineering work they do.
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