UF researchers develop improved gene therapy agent

Replacing one amino acid on the surface of a virus that shepherds corrective genes into cells could be the breakthrough scientists have needed to make gene therapy a more viable option for treating genetic diseases such as hemophilia, University of Florida researchers say.

Reporting in the journal Proceedings of the National Academy of Sciences today (May 19), UF geneticists say they have developed a new version of the adeno-associated virus used in gene therapy that works about 30 times more efficiently in mice than vectors scientists currently rely on.

EurekAlert!