UMass Medical School scientists honored for gene therapy patent for alpha 1 antitrypsin deficiency

UMass Medical School Communications

October 25, 2017

Scientists from UMass Medical School were honored by the Boston Patent Law Association on Monday, Oct. 23, for a patent that describes an improved method of using gene therapy to treat alpha-1 antitrypsin deficiency.

Terence R. Flotte, MD, the Celia and Isaac Haidak Professor of Medical Education, executive deputy chancellor, provost and dean of the School of Medicine; Phillip D. Zamore, PhD, the Gretchen Stone Cook Chair in the Biomedical Sciences, and chair and professor of the RNA Therapeutics Institute; and Christian Mueller, PhD, associate professor of pediatrics, were recognized for the patent. Alpha-1 antitrypsin deficiency is an inherited disease that causes liver and lung damage. Currently, there is no curative treatment for the disease; many people with the disease manage the symptoms with intravenous infusions of alpha-1 antitrypsin purified from donated human plasma. New clinical approaches under development at UMass Medical School use gene therapy and gene editing to form the basis of a treatment to remove the source of the toxic protein in the liver, while ramping up production of healthy alpha-1 proteins.

The purpose of the Boston Patent Law Association’s Invented Here! Program is to celebrate New England innovators, their inventions and the stories behind their innovations. Through the program, the Boston Patent Law Association aspires to change the nation’s relationship with science and technology and provides educational opportunities that inform the public about local innovators and innovations.

Since its inception in 2010, the program has recognized 49 inventions and more than 170 inventors.

Related story on UMassMedNow:
New gene editing approach for alpha-1 antitrypsin deficiency shows promise in UMMS study

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