%0 Journal Article
%A Lansing, Felix
%A Mukhametzyanova, Liliya
%A Rojo-Romanos, Teresa
%A Iwasawa, Kentaro
%A Kimura, Masaki
%A Paszkowski-Rogacz, Maciej
%A Karpinski, Janet
%A Grass, Tobias
%A Sonntag, Jan
%A Schneider, Paul Martin
%A Günes, Ceren
%A Hoersten, Jenna
%A Schmitt, Lukas Theo
%A Rodriguez-Muela, Natalia
%A Knöfler, Ralf
%A Takebe, Takanori
%A Buchholz, Frank
%T Correction of a Factor VIII genomic inversion with designer-recombinases.
%J Nature Communications
%V 13
%N 1
%@ 2041-1723
%C [London]
%I Nature Publishing Group UK
%M DZNE-2022-00242
%P 422
%D 2022
%X Despite advances in nuclease-based genome editing technologies, correcting human disease-causing genomic inversions remains a challenge. Here, we describe the potential use of a recombinase-based system to correct the 140 kb inversion of the F8 gene frequently found in patients diagnosed with severe Hemophilia A. Employing substrate-linked directed molecular evolution, we develop a coupled heterodimeric recombinase system (RecF8) achieving 30
%K Amino Acid Sequence
%K Base Sequence
%K Cell Differentiation
%K Chromosome Inversion: genetics
%K Clone Cells
%K Directed Molecular Evolution
%K Endothelial Cells: cytology
%K Endothelial Cells: metabolism
%K Exons: genetics
%K Factor VIII: genetics
%K HEK293 Cells
%K HeLa Cells
%K Humans
%K Induced Pluripotent Stem Cells: metabolism
%K Inverted Repeat Sequences: genetics
%K Recombinases: metabolism
%K Recombination, Genetic: genetics
%K Substrate Specificity
%K Whole Genome Sequencing
%K Recombinases (NLM Chemicals)
%K Factor VIII (NLM Chemicals)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:35058465
%2 pmc:PMC8776779
%R 10.1038/s41467-022-28080-7
%U https://pub.dzne.de/record/163482