Base editing corrects alpha-1 antitrypsin gene defect in a patient's liver, case report shows
A one-off infusion of an experimental base-editing therapy corrected more than half of the faulty gene copies in a 66-year-old man's liver. The peer-reviewed evidence so far is that single case.
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Doctors at Hannover Medical School (MHH) in Germany and Ren Ji Hospital in Shanghai have reported that an experimental gene-editing treatment corrected the genetic error behind alpha-1 antitrypsin deficiency in a patient's liver. The peer-reviewed evidence is a single-patient case report in the Journal of Hepatology, published online on 2 November 2026.
How this story was sourced.
6 sources from 6 organisations, all of them official or primary documents.
Official and primary documents 6
The body that made the decision, the paper, or the organisation’s own release.
- Journal of Hepatology Liver biopsy confirms precise and efficient correction of SERPINA1 after in vivo base editing in a patient with alpha-1 antitrypsin deficiency journal-of-hepatology.eu
- PubMed abstract of the Journal of Hepatology case report pubmed.ncbi.nlm.nih.gov
- Elsevier via EurekAlert Gene correction therapy successfully treats alpha-1 antitrypsin deficiency in vivo eurekalert.org
- Hannover Medical School via idw press release on base editing for alpha-1 antitrypsin deficiency idw-online.de
- YolTech proof-of-concept data for YOLT-202 at the European Respiratory Society Congress 2026 yoltx.com
- ClinicalTrials.gov Study of YOLT-202 in the Treatment of Alpha-1 Antitrypsin Deficiency (NCT07193615) clinicaltrials.gov
The sources as the story lists them
Sources
- Journal of Hepatology: Liver biopsy confirms precise and efficient correction of SERPINA1 after in vivo base editing in a patient with alpha-1 antitrypsin deficiency02935-1/fulltext)
- PubMed: abstract of the Journal of Hepatology case report
- Elsevier via EurekAlert: Gene correction therapy successfully treats alpha-1 antitrypsin deficiency in vivo
- Hannover Medical School via idw: press release on base editing for alpha-1 antitrypsin deficiency
- YolTech: proof-of-concept data for YOLT-202 at the European Respiratory Society Congress 2026
- ClinicalTrials.gov: Study of YOLT-202 in the Treatment of Alpha-1 Antitrypsin Deficiency (NCT07193615)
The story so far. More on Gene editing, Health and Rare disease.
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