With an eye on safety, UF experts explore cellular mechanisms of ge...( rapy can work but where it's not likel...)

The scientists discovered the protein's role in the process by comparing gene therapy in two strains of mice. A mutation in one of the strains prevents mice from producing the protein.

"In the absence of this protein, over time, much of newly inserted DNA integrates into the host cell's chromosome. Theoretically, that's the kind of event that in some small percentage of cases would be harmful," Flotte said.

"But in the presence of this protein, the new DNA stayed in this independent string form. We saw no detectable integrations. These experiments were conducted in mice, rather than people, but from these mouse models we've been able to get a very good understanding of cellular mechanisms of gene therapy."

Song noted that in other experiments using human cell cultures, the protein also prevented AAV from integrating into a chromosome.

Dr. Kenneth I. Berns, UF's vice president for health affairs and an early pioneer of using AAV as a gene therapy vector, and Philip J. Laipis, a professor and associate chairman of the College of Medicine's biochemistry and molecular biology department, also contributed to the research effort.

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Contact: Victoria White
vjwhite@tampabay.rr.com
352/344-2738
University of Florida
5-Apr-2001