Correction of a Factor VIII genomic inversion with designer-recombinases
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.
| Main Authors: | , , , , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Portfolio
2022-01-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-022-28080-7 |
| _version_ | 1818954051024322560 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Felix Lansing |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-20T10:16:01Z |
| format | Article |
| id | doaj.art-8d46d2035aed42e78c73c8d76348317f |
| institution | Directory Open Access Journal |
| issn | 2041-1723 |
| language | English |
| last_indexed | 2024-12-20T10:16:01Z |
| publishDate | 2022-01-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Nature Communications |
| spelling | doaj.art-8d46d2035aed42e78c73c8d76348317f2022-12-21T19:44:04ZengNature PortfolioNature Communications2041-17232022-01-0113111510.1038/s41467-022-28080-7Correction of a Factor VIII genomic inversion with designer-recombinasesFelix Lansing0Liliya Mukhametzyanova1Teresa Rojo-Romanos2Kentaro Iwasawa3Masaki Kimura4Maciej Paszkowski-Rogacz5Janet Karpinski6Tobias Grass7Jan Sonntag8Paul Martin Schneider9Ceren Günes10Jenna Hoersten11Lukas Theo Schmitt12Natalia Rodriguez-Muela13Ralf Knöfler14Takanori Takebe15Frank Buchholz16Medical Systems Biology, Medical Faculty, Technical University DresdenMedical Systems Biology, Medical Faculty, Technical University DresdenMedical Systems Biology, Medical Faculty, Technical University DresdenDivision of Gastroenterology, Hepatology and Nutrition, Division of Developmental Biology, Center for Stem Cell and Organoid Medicine (CuSTOM) Cincinnati Children’s Hospital Medical CenterDivision of Gastroenterology, Hepatology and Nutrition, Division of Developmental Biology, Center for Stem Cell and Organoid Medicine (CuSTOM) Cincinnati Children’s Hospital Medical CenterMedical Systems Biology, Medical Faculty, Technical University DresdenMedical Systems Biology, Medical Faculty, Technical University DresdenMedical Systems Biology, Medical Faculty, Technical University DresdenMedical Systems Biology, Medical Faculty, Technical University DresdenMedical Systems Biology, Medical Faculty, Technical University DresdenDepartment of Cell and Developmental Biology, Max Planck Institute for Molecular BiomedicineMedical Systems Biology, Medical Faculty, Technical University DresdenMedical Systems Biology, Medical Faculty, Technical University DresdenGerman Center for Neurodegenerative Diseases, Helmholtz AssociationDepartment of Pediatric Hematology and Oncology, University Hospital DresdenDivision of Gastroenterology, Hepatology and Nutrition, Division of Developmental Biology, Center for Stem Cell and Organoid Medicine (CuSTOM) Cincinnati Children’s Hospital Medical CenterMedical Systems Biology, Medical Faculty, Technical University DresdenCorrection 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.https://doi.org/10.1038/s41467-022-28080-7 |
| spellingShingle | 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 Correction of a Factor VIII genomic inversion with designer-recombinases Nature Communications |
| title | Correction of a Factor VIII genomic inversion with designer-recombinases |
| title_full | Correction of a Factor VIII genomic inversion with designer-recombinases |
| title_fullStr | Correction of a Factor VIII genomic inversion with designer-recombinases |
| title_full_unstemmed | Correction of a Factor VIII genomic inversion with designer-recombinases |
| title_short | Correction of a Factor VIII genomic inversion with designer-recombinases |
| title_sort | correction of a factor viii genomic inversion with designer recombinases |
| url | https://doi.org/10.1038/s41467-022-28080-7 |
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