Article

A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models

2025-05-09

Abstract excerpt

Alpha-1 antitrypsin deficiency (AATD) is commonly caused by a G-to-A mutation in the SERPINA1 gene (the PiZ mutation). The mutant PiZ AAT protein is sequestered in hepatocytes, causing lung emphysema due to insufficient AAT protein to inhibit neutrophil elastase in the lung. Here we show that a compact adenine base editor (ABE) with an evolved Cas9 nickase derived from Neisseria meningitidis (eNme2.C) can be packa...

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Literature Corpus work: a3c137ae-824b-59ce-9f48-0ed3a1eb1152
DOI: 10.1101/2025.05.07.652636

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