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New Research Holds Promise for Gene Engineering and Therapy
Date:4/4/2011

NEW YORK, April 4, 2011 /PRNewswire/ -- Scientists at Polytechnic Institute of New York University (NYU-Poly) and at the NYU College of Dentistry have discovered a biochemical version of a principle well known among confectioners. Call it the "peanut butter and chocolate" rule:  Sometimes two things work better together than alone.

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Seiichi Yamano, assistant professor in the Department of Prosthodontics at NYU's College of Dentistry, and Jin Kim Montclare, who runs NYU-Poly's Protein Engineering and Molecular Design Lab, have developed a remarkably effective way to combine two methods that scientists use as vehicles to carry DNA into cell nuclei. The result could help researchers understand gene function, analyze proteins and ultimately improve gene therapy for a number of genetic diseases like hemophilia and muscular dystrophy, acquired diseases like cancer, neurodegenerative diseases like ALS, as well as HIV and hepatitis.

Their research aims to improve the efficiency of a procedure called transfection, the artificial introduction of genetic material into cells by means of a non-viral "vector," essentially a biochemical courier.

But transfection is difficult because of the sheer size and the electric charge of the DNA that must penetrate the cell's membranes.

Indeed, "convincing" a cell to usher a genetic macromolecule to its inner sanctum is a bit like trying to walk a grand piano through airport security. Even if the macromolecules make it to a cell's cytoplasm, the cell itself sets up a gauntlet of barriers to transfection.

In the past, most transfection vehicles were essentially decommissioned viruses; they could be engineered to carry any sort of genetic ma
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SOURCE Polytechnic Institute of New York University
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