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UD professor working to use gene therapy to treat blood disorders

February 15, 2020  |  Delaware Public Media

A University of Delaware professor is receiving grant funding to develop a new therapy for people with low blood platelet counts and other blood disorders.

When patients don’t have enough blood platelets, they can’t form clots and this can sometimes be a lethal condition. These patients rely on platelet donations as a treatment, but those are often in short supply since they cannot be frozen and are only good for a few days.

 

A few years ago, UD’s professor of Chemical and Biomolecular Engineering Terry Papoutsakis discovered a way to get patients to produce more blood platelets in their own bloodstream by introducing microparticles made from cultured stem cells.

 

These microparticles can be frozen to be used on a large scale. And Papoutsakis says they could also be used to treat genetic blood disorders like primary immune deficiencies.

 

“To be able to use those particles for applications in gene therapy for a variety of blood diseases—a lot of genetic diseases or acquired diseases for patients,” said Papoutsakis. “So that’s a difficult problem to deal with and we think this has great promise.”       

Papoutsakis is being awarded $250,000 through a partnership between University City Science Center and CSL Behring meant to find new biotherapies. He says the award took a few years to secure as a result of contract negotiations around intellectual property rights.

 

“It took a little while to do it, but that is to be understood that it would, because of the potential that this might develop into something quite bigger than we currently see,” he said. 

     

Papoutsakis says he has already had some success testing his discovery on mice. He says the award will fund a continuation of those tests, moving the therapy closer to clinical trials on human patients.