Pinpoint targeting promises improved success in gene therapy for blood and other disorders

CHAPEL HILL - Research on a rare inherited blood disorder known as Glanzmann's disease shows for the first time that it's possible to target specific types of cells during gene therapy and avoid the less effective -- and possibly more dangerous -- "shotgun" approach now used, according to a new University of North Carolina at Chapel Hill School of Medicine study.

With more work, the method could help treat a host of gene-related illnesses including various cancers, UNC-CH scientists say.

"Using techniques first developed by other investigators, we created a way of targeting the expression, or production, of genetic material in blood platelets without affecting red or white blood cells at the same time," said Dr. Gilbert C. White II, professor of medicine and director of UNC-CH's Center for Thrombosis and Hemostasis. "This offers great promise for improved treatment of inherited blood disorders such as hemophilia and conceptual support for using the approach for non-inherited illnesses such as cancer.

"For example, if you wanted to get poison genes into cancer cells but not into other healthy cells, one could attach the poison gene to specific promoters -- or pieces of DNA - that function only in cancer cells and not in others."

A report on the study appears in the Aug. 17 issue of the Proceedings of the National Academy of Sciences. Besides White, authors are Drs. David A. Wilcox, a former UNC-CH postdoctoral fellow now at the Medical College of Wisconsin; John C. Olsen, research assistant professor of medicine at UNC-CH; and Lori Ishizawa and Michael Griffith of Nexell Therapeutics Inc. of Irvine, Calif.

Wilcox said he and his colleagues concentrated on correcting primitive stem cells - sometimes considered "parent" cells -- capable of developing into more specialized large cells. Those younger monster cells, called megakaryocytes, form in bone marrow and eventually shatter into at least hundr

Contact: David Williamson
University of North Carolina at Chapel Hill

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