Genetic & Genomic MedicinemedRxivPreprint
Liver biopsy confirms precise and efficient correction of SERPINA1 after in vivo Base Editing in a Patient with Alpha-1 Antitrypsin Deficiency
Simon Alexander Krooss, Taihua Yang, Qinggong Yuan…·Michael Ott
Liver biopsy after PI*ZZ gene correction therapy reveals efficient and accurate base editing.
DOI 10.64898/2026.06.01.26354551