University of Florida researchers test drug from 'transgenic sheep' in study at Shands at UF teaching hospital

GAINESVILLE---In an early test of breakthrough technology, University of Florida researchers have begun the first pilot study to determine whether the milk of genetically modified sheep can help people prone to life-threatening lung problems caused by a specific protein deficiency.

PPL Therapeutics of Edinburgh, Scotland, the lead company involved in the development of Dolly the cloned sheep, uses recombinant DNA technology -- in which a gene from one organism is inserted into the DNA of another -- to create "transgenic sheep" that produce the human protein alpha-1-antitrypsin, or AAT. The protein is then extracted from the milk.

Within the past 10 days, six patients whose bodies manufacture inadequate amounts of AAT, leaving them vulnerable to emphysema, have begun receiving inhaled doses of it at Shands at UF teaching hospital.

If the technique proves successful in this and larger controlled trials, a vast new source of AAT, at a sharply reduced price, potentially would be available to treat the estimated 100,000 Americans who have a deficiency of the protective protein that helps to keep lung inflammation and damage in check.

Such a new supply is critical: Currently, only a limited amount is available through blood plasma donations, so many eligible candidates must do without the therapy, which is delivered intravenously at an estimated cost of $60,000 to $80,000 annually per patient.

"I have great hopes that this will be the cornerstone therapy for this disease," said Dr. Mark Brantly, lead investigator for the study and a professor of medicine, and molecular genetics and microbiology at UF's College of Medicine.

"It could be the major 'bridge' treatment before we advance to gene therapy for this disorder," said Brantly, who also is affiliated with UF's new Genetics Institute, which is exploring such possibilities with grant support from the National Institutes of Health.

"One of the primary reasons for creating the Genetics Insti

Contact: Vicki White
University of Florida

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