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UMass Medical School study by Mueller lab finds promise in "nuclease-free" gene editing for alpha-1 antitrypsin deficiency.

Scientists from UMass Medical School were honored by the Boston Patent Law Association for an improved method of using gene therapy to treat alpha-1 antitrypsin deficiency.

Scott E. Kopec, MD, and Christian Mueller, PhD, were given the Shillelagh award by the Alpha-1 Foundation.

Researchers at the UMass Medical School have been commissioned by the Alpha-1 Project to develop a PiZ antibody, an essential tool for testing potential therapies for alpha-1 antitrypsin deficiency.

Data from a study by Terence Flotte and UMass Medical School colleagues demonstrates sustained protein expression 5 years after a gene therapy for AAT deficiency.

Program project grant will develop new therapies for alpha-1 antitrypsin deficiency, a debilitating lung disease.

Research at UMMS offers new evidence that a dual therapy approach for patients with alpha-1 antitrypsin deficiency may have potential for simultaneously treating the lung and liver diseases commonly associated with the hereditary disorder.

Researchers from UMass Chan Medical School have received a five-year $13.6 million program project grant from the National Heart, Lung and Blood Institute to develop new gene therapy models for alpha-1 antitrypsin deficiency, a chronic, debilitating genetic lung disease that shortens the lifespan.

Past milestones and future treatment prospects will be described by world-renowned Alpha-1 researchers at a free program at UMMS on Saturday, April 20.

Researchers at the University of Massachusetts Amherst and UMass Chan Medical School mapped the expression and maturation of the protein alpha-1 antitrypsin (AAT) with unprecedented clarity. UMass Chan gene therapy pioneer Terence R. Flotte, MD, is a co-author on the paper.