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Gene Therapy Shows Promise Against Hereditary Lung Disease

An 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// patients and shows signs of being effective, University of Florida researchers say.

In a clinical trial, researchers evaluated the safety of using a so-called gene vector - in this case an adeno-associated virus - to deliver a corrective gene to 12 patients who are unable to produce a protein essential for health called alpha-1 antitrypsin.

“The primary end point in the trial was to see whether it was safe to give patients this gene transfer vector and then 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,” said Terence Flotte, M.D., a pediatrician, geneticist and microbiologist with UF’s College of Medicine and a member of the Powell Gene Therapy Center and the UF Genetics Institute. “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. 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 today (Nov. 21) in the journal Human Gene Therapy.

Physicians injected doses of the virus containing copies of the gene for alpha-1 antitrypsin into the patients’ upper arms. Essentially, the virus is intended to “infect” patients’ cells with replacement genes that will do the necessary work to produce alpha-1 protein. UF scientists have successfully developed the technique in animal models.

The next step is to test the therapy with a different version of the adeno-associated virus; about 200 variations of the virus exist in nature.

“We have another version of the virus that appears in animal studies to be close to a thousandfold more pot
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