Article

Liver biopsy confirms precise and efficient correction of SERPINA1 after <i>in vivo</i> Base Editing in a Patient with Alpha-1 Antitrypsin Deficiency

2026-06-09

Abstract excerpt

<h4>Background</h4> Alpha-1 antitrypsin deficiency (AATD) caused by the PI*ZZ mutation (Glu342Lys) results in hepatic accumulation of misfolded AAT-Z protein and reduced circulating AAT levels, leading to progressive liver disease and emphysema. Gene correction therapy represents a potentially curative approach by directly correcting the underlying genetic defect. We report the first case of successful hepatic ge...

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Literature Corpus work: a5118c59-1e74-5732-9d48-85640b7f3ae1
DOI: 10.64898/2026.06.01.26354551

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