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, Nora Drick, Malte Sgodda, Julia Held, Patrick Behrendt, Björn Hartleben, Rembert Koczulla, Xiaoying Ma, Ying Liu, Heiner Wedemeyer, Sabina M Janciauskiene, Nataliya Di Donato, Tobias Cantz, , Michael Ott
Liver biopsy after PI*ZZ gene correction therapy reveals efficient and accurate base editing.
medRxiv · Jun 9, 2026 · PreprintGenetic & Genomic Medicine