Voice-over:
About one-hundred thousand Americans have alphaǃ antitrypsin deficiency
or alphaǃ- a debilitating lung disease that can cause emphysema and greatly shorten a
patient's lifespan.
Voice-over:
Although alphaǃ is among the most common hereditary disorders, few treatments exist.
But University of Florida researchers have completed the first study to inject alphaǃ patients with a harmless virus to deliver copies of an important gene they're missing. Twelve patients with the lung disorder got injections in the upper arm muscle. Follow-up showed that this method was safe. And although scientists weren't testing for therapeutic effects, they saw signs in one of the twelve patients that the corrective gene was effectively transferred to the patient.
Dr. Terry Flotte / UF genetics researcher
"The primary end point in the trial was to see if it was safe to give patients this gene transfer vector and then also to try to begin to see if we could get the dose into a range where we would begin to replace the missing protein in the blood."
Voice-over:
Achieving the goal of establishing long-term therapy using gene transfer will require more tests. But doctors say this study shows that appropriate treatment levels of the gene may be obtainable in the blood. UF researchers now plan to continue their studies in people, testing more efficient versions of the same gene delivery agent, or vector. The hope? That this approach could someday deliver long-lasting results.
Dr. Terry Flotte / UF genetics researcher
"This really sets us up well to focus on improving the vector, the efficiency of transfer of the gene, so that we can get more kick if you will from the gene transfer vector."
Voice-over:
At the University of Florida Health Science Center, I'm Mike Garrison.
