Synthetic protein could have applications in Gaucher as well as asthma

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New York University researchers seeking to prove how a protein protects asthmatics from lung damage may have found a new treatment for Gaucher disease, in which fat accumulates in cells and certain organs.

Gaucher disease affects as many as one in every 50,000 people in the U.S. and as many as one in 500 people of Ashkenazi Jewish heritage, according to NIH estimates. Its symptoms include fatty buildup in and enlargement of the liver and spleen, joint pain, easy bruising and lung disease. It is linked to low  production of the protein progranulin and a deficiency in the enzyme GBA, which breaks down the fat glucocerebroside. In patients with Gaucher, GBA tends to build up outside lysosomes rather than inside them where it is needed.

Researchers from the NYU Langone Medical Center conducted a set of experiments in mice that produced abnormally low amounts of progranulin, according to a statement. They found that delivering synthetic progranulin, or Pcgin, reduced lung tissue swelling by as much as 60%. But they also discovered that Pcgin reversed “most effects” of Gaucher disease in mouse and human cell studies. Current Gaucher treatments include enzyme replacement therapy.

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"Our results suggest a new way to treat Gaucher's disease that corrects abnormal enzyme delivery by progranulin to lysosomes, as opposed to current treatment strategies that temporarily replenish lysosomal GBA stores, which are then steadily consumed," said senior study investigator Chuanju Liu, a professor in the Departments of Orthopaedic Surgery and Cell Biology at NYU Langone, in the statement.

They found that adding Pcgin to blood cells taken from Gaucher patients resulted in 40% less GBA clumping within a week, according to the statement.

NYU Langone and the research team hold a patent on related potential therapies. More research is still needed to ascertain how, exactly, progranulin reduces cell swelling in asthma patients, they said in the statement. This could also turn up new insights on the protein’s role in Gaucher.