Gene Therapy Trial Termed A Success

A U.S. trial evaluating the safety of using a gene vector to deliver a corrective gene to patients with a common hereditary disorder has been completed.

The experimental gene therapy to combat alpha-1 antitrypsin deficiency -- a common hereditary disorder that causes lung and liver disease -- has caused no harmful effects in the 12 patients and shows signs of being effective, University of Florida researchers said.

"The primary end point in the trial was to see whether it was safe to give patients this gene transfer vector ...," said Terence Flotte, a pediatrician, geneticist and microbiologist with the University of Florida 's College of Medicine.

"We found that we can use this agent safely and we also saw evidence in the patients' blood that the higher doses successfully introduced the vector DNA," said Flotte. "In one patient we saw evidence for a very brief period that some of the alpha-1 protein was being produced but not at a high enough level to be beneficial."

The findings appear online in the journal Human Gene Therapy.