Nature Communications (Jan 2022)
Correction of a Factor VIII genomic inversion with designer-recombinases
- 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
Affiliations
- Felix Lansing
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- Liliya Mukhametzyanova
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- Teresa Rojo-Romanos
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- Kentaro Iwasawa
- Division of Gastroenterology, Hepatology and Nutrition, Division of Developmental Biology, Center for Stem Cell and Organoid Medicine (CuSTOM) Cincinnati Children’s Hospital Medical Center
- Masaki Kimura
- Division of Gastroenterology, Hepatology and Nutrition, Division of Developmental Biology, Center for Stem Cell and Organoid Medicine (CuSTOM) Cincinnati Children’s Hospital Medical Center
- Maciej Paszkowski-Rogacz
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- Janet Karpinski
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- Tobias Grass
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- Jan Sonntag
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- Paul Martin Schneider
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- Ceren Günes
- Department of Cell and Developmental Biology, Max Planck Institute for Molecular Biomedicine
- Jenna Hoersten
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- Lukas Theo Schmitt
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- Natalia Rodriguez-Muela
- German Center for Neurodegenerative Diseases, Helmholtz Association
- Ralf Knöfler
- Department of Pediatric Hematology and Oncology, University Hospital Dresden
- Takanori Takebe
- Division of Gastroenterology, Hepatology and Nutrition, Division of Developmental Biology, Center for Stem Cell and Organoid Medicine (CuSTOM) Cincinnati Children’s Hospital Medical Center
- Frank Buchholz
- Medical Systems Biology, Medical Faculty, Technical University Dresden
- DOI
- https://doi.org/10.1038/s41467-022-28080-7
- Journal volume & issue
-
Vol. 13,
no. 1
pp. 1 – 15
Abstract
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.
