Correction of a Factor VIII genomic inversion with designer-recombinases

oleh: Felix Lansing, Liliya Mukhametzyanova, Teresa Rojo-Romanos, Kentaro Iwasawa, Masaki Kimura, Maciej Paszkowski-Rogacz, Janet Karpinski, Tobias Grass, Jan Sonntag, Paul Martin Schneider, Ceren Günes, Jenna Hoersten, Lukas Theo Schmitt, Natalia Rodriguez-Muela, Ralf Knöfler, Takanori Takebe, Frank Buchholz

Format: Article
Diterbitkan: Nature Portfolio 2022-01-01

Deskripsi

Correction of disease-causing large genomic inversions remains challenging. Here, the authors developed a dual designer-recombinase system (RecF8) that efficiently corrects a 140 kb inversion frequently found in patients with severe Hemophilia A.