Tag: <span>Antitrypsin Deficiency</span>

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Second generation gene therapy for alpha 1-antitrypsin deficiency
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Second generation gene therapy for alpha 1-antitrypsin deficiency

MARY ANN LIEBERT, INC./GENETIC ENGINEERING NEWS IMAGE: HUMAN GENE THERAPY CREDIT: MARY ANN LIEBERT, INC., PUBLISHERS Researchers report on the safety of a gene therapy to treat the common autosomal recessive hereditary disorder alpha 1-antitrypsin (AAT) deficiency in a new article in the peer-reviewed journal Human Gene Therapy. In ATT deficiency, neutrophil proteases destroy the...

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New Gene Editing Approach for Alpha-1 Antitrypsin Deficiency Shows Promise in UMMS Study

WORCESTER — A new study by scientists at UMass Medical School shows that using a technique called “nuclease-free” gene editing to correct cells with the mutation that causes a rare liver disease leads to repopulation of the diseased liver with healthy cells. Alpha-1 antitrypsin deficiency is an inherited disease that causes liver and lung damage;...