News
Foundation, Talecris announce two research grant awards
Researchers to develop line of Alpha-1 mice, map structure of AAT polymers
RESEARCH TRIANGLE PARK, NC and MIAMI, FL — The Alpha-1 Foundation has awarded two new research grants for projects at the University of Florida and Brandeis University. The grants were made possible through an unrestricted charitable donation from Talecris Biotherapeutics to the Alpha-1 Foundation.
The first grant, given to Sihong Song, PhD, of the University of Florida, will enable him to develop a line of mice with Alpha-1 Antitrypsin Deficiency.
No mouse models currently exist for studying the functions of the alpha-1-antitrypsin (AAT) protein. Song will use genetic engineering techniques to replace the gene for alpha-1 protein in the mice being studied. The goal is to develop a line of transgenic mice which express the mutant “Z” alpha-1 protein – mice with Alpha-1.
The Alpha-1 mice will be studied for the development of lung and liver disease. “We hope that these AAT-deficient mouse models will reveal novel functions of AAT and be useful in evaluating treatments for AAT-related human diseases,” said Song.
The second grant, to Brandeis University in Waltham, MA, will be used to fund the work of Anne Gershenson, PhD, who plans to map the structure of AAT to aid in designing new treatments for Alpha-1.
AAT, the alpha-1 protein, is mainly made in the liver. Normal AAT is a single protein called a monomer. In people living with Alpha-1, the single AAT proteins link together to form long chains called polymers. The long polymer chains cannot be transported normally into the bloodstream, so a deficiency of AAT develops in the blood. This is considered a root cause of lung disease in Alpha-1. The AAT polymer chains accumulate in the liver cells, and in some Alpha-1 patients, this leads to liver disease.
Gershenson’s study will use precise measuring and mapping techniques to attempt to determine the molecular structure of AAT polymers.
