Correction of a Factor VIII genomic inversion with designer-recombinases
Felix Lansing, Liliya Rinatovna Mukhametzyanova, Teresa Rojo Romanos, Kentaro Iwasawa, Masaki Kimura, Maciej Paszkowski‐Rogacz, Janet Karpinski, Tobias Graß, Jan Sonntag, Paul Martin Schneider, Ceren Güneş, Jenna Hoersten, Lukas Theo Schmitt, Natalia Rodríguez‐Muela, Ralf Knöfler, , Frank Buchholz
This study corrects a 140 kb genomic inversion in the F8 gene using a designer recombinase system, restoring Factor VIII mRNA expression in patient-derived cells.
Nature Communications · Jan 20, 2022Peer-reviewedMolecular Biology