Molecular BiologyNature CommunicationsPeer-reviewed
Correction of a Factor VIII genomic inversion with designer-recombinases
Felix Lansing, Liliya Rinatovna Mukhametzyanova, Teresa Rojo Romanos…·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.
30 citations