New Technology Makes Gene Therapy More Effective

November 10, 1998

Better Weapon to Fight Liver Disease, Sickle Cell Anemia, Other Genetic Diseases

CHICAGO, November 10 -- Research that could point the way to more effective gene therapies for liver and other diseases was made public at The American Association for the Study of Liver Diseases 1998 Annual Meeting in Chicago, November 6 - 10.

Betsy T. Kren, PhD, of the University of Minnesota Medical School reported on a study of a new technology that permanently corrects genetic defects caused by what are called "single point mutations" in the liver cells of laboratory rats. Most gene therapy uses viruses to deliver altered bits of DNA to specific genes to correct defective DNA. But viruses have limited effectiveness, in part because they deliver DNA randomly to the genes. In the study, researchers used a new technology to replace the bad DNA in liver cells with new healthy DNA much more effectively and efficiently.

According to Clifford Steer, MD, head of the project, researchers put altered bits of DNA into specially designed targeting molecules and injected the molecules into tail veins of rats. Those bits of DNA made their way to the nuclei of genetically damaged liver cells. Through a complicated process, the DNA bits "tricked" the damaged cells into precisely correcting the specific defective nucleotide in the DNA that the researchers had targeted.

"The potential future impact of this technology is profound," says Steer. "Most genetic diseases of the liver are single point mutations, as are sickle cell and many other non-liver diseases."
AASLD is the leading medical organization for liver researchers and physicians. Founded by physicians in 1950, AASLD's mission is to advance the science and practice of hepatology. Today, AASLD provides representation and education for more than 2,200 hepatologists worldwide.

American Association For The Study of Liver Diseases

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