Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency—a promising leap toward treating humans with this rare inherited disease.
Jul 20, 2025
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National Jewish Health researchers with the Advanced Diagnostic Laboratories have developed a molecular diagnostic test capable of accurately diagnosing a major genetic cause of chronic obstructive pulmonary disease (COPD) ...
May 7, 2025
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Acquired DNA mutations found in the SERPINA1 gene can protect liver cells from damage in patients with alpha-1 antitrypsin deficiency, new research shows. Alpha-1 antitrypsin deficiency (A1AD) is a genetic condition caused ...
Mar 10, 2025
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A multicenter safety study at UC Davis Health tested a protein drug (INBRX-101), developed to improve care for people with alpha-1 antitrypsin deficiency (AATD), and showed the therapy is quite safe.
Jul 11, 2024
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Patients with lung disease are more likely to develop heart disease independent of any risk factors, according to new research.
Mar 22, 2024
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Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of Medicine and Health Sciences. ...
Nov 1, 2023
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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 Human Gene Therapy.
Feb 22, 2023
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Alpha-1 antitrypsin deficiency (AATD) is an important and under-recognized genetic cause of liver disease. The most common mutation associated with liver disease results from a single change within the DNA sequence known ...
Dec 6, 2022
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In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass Chan Medical School recently announced that ...
Jul 25, 2022
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Researchers at Saint Louis University's School of Medicine, in collaboration with Arrowhead Pharmaceuticals and Takeda Pharmaceuticals, report the first effective drug to treat a rare, genetic liver disease that formerly ...
Jul 11, 2022
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