In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice
Hemochromatosis is a metabolic disorder caused by mutations in the HFE gene. Here, the authors show that a single administration of AAV8 vectors expressing an Adenine Base Editor facilitates efficient in vivo gene correction in hepatocytes and leads to improvement of iron-specific parameters in the...
| Main Authors: | , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Portfolio
2022-09-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-022-32906-9 |
| _version_ | 1798003038395301888 |
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| author | Alice Rovai BoMee Chung Qingluan Hu Sebastian Hook Qinggong Yuan Tibor Kempf Florian Schmidt Dirk Grimm Steven R. Talbot Lars Steinbrück Jasper Götting Jens Bohne Simon A. Krooss Michael Ott |
| author_facet | Alice Rovai BoMee Chung Qingluan Hu Sebastian Hook Qinggong Yuan Tibor Kempf Florian Schmidt Dirk Grimm Steven R. Talbot Lars Steinbrück Jasper Götting Jens Bohne Simon A. Krooss Michael Ott |
| author_sort | Alice Rovai |
| collection | DOAJ |
| description | Hemochromatosis is a metabolic disorder caused by mutations in the HFE gene. Here, the authors show that a single administration of AAV8 vectors expressing an Adenine Base Editor facilitates efficient in vivo gene correction in hepatocytes and leads to improvement of iron-specific parameters in the liver and the blood in mouse models of the disease. |
| first_indexed | 2024-04-11T12:01:40Z |
| format | Article |
| id | doaj.art-41baa3360d14427b871c13b614264a6d |
| institution | Directory Open Access Journal |
| issn | 2041-1723 |
| language | English |
| last_indexed | 2024-04-11T12:01:40Z |
| publishDate | 2022-09-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Nature Communications |
| spelling | doaj.art-41baa3360d14427b871c13b614264a6d2022-12-22T04:24:50ZengNature PortfolioNature Communications2041-17232022-09-0113111010.1038/s41467-022-32906-9In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis miceAlice Rovai0BoMee Chung1Qingluan Hu2Sebastian Hook3Qinggong Yuan4Tibor Kempf5Florian Schmidt6Dirk Grimm7Steven R. Talbot8Lars Steinbrück9Jasper Götting10Jens Bohne11Simon A. Krooss12Michael Ott13Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical SchoolDepartment of Cardiology and Angiology, Hannover Medical SchoolDepartment of Gastroenterology, Hepatology and Endocrinology, Hannover Medical SchoolDepartment of Gastroenterology, Hepatology and Endocrinology, Hannover Medical SchoolDepartment of Gastroenterology, Hepatology and Endocrinology, Hannover Medical SchoolDepartment of Cardiology and Angiology, Hannover Medical SchoolDepartment of Biosystems Science and Engineering, ETH ZürichDepartment of Infectious Diseases/Virology, Medical Faculty, University of Heidelberg, Cluster of Excellence CellNetworksInstitute for Laboratory Animal Science and Central Animal Facility, Hannover Medical SchoolInstitute of Virology, Hannover Medical SchoolInstitute of Virology, Hannover Medical SchoolInstitute of Virology, Hannover Medical SchoolDepartment of Gastroenterology, Hepatology and Endocrinology, Hannover Medical SchoolDepartment of Gastroenterology, Hepatology and Endocrinology, Hannover Medical SchoolHemochromatosis is a metabolic disorder caused by mutations in the HFE gene. Here, the authors show that a single administration of AAV8 vectors expressing an Adenine Base Editor facilitates efficient in vivo gene correction in hepatocytes and leads to improvement of iron-specific parameters in the liver and the blood in mouse models of the disease.https://doi.org/10.1038/s41467-022-32906-9 |
| spellingShingle | Alice Rovai BoMee Chung Qingluan Hu Sebastian Hook Qinggong Yuan Tibor Kempf Florian Schmidt Dirk Grimm Steven R. Talbot Lars Steinbrück Jasper Götting Jens Bohne Simon A. Krooss Michael Ott In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice Nature Communications |
| title | In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice |
| title_full | In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice |
| title_fullStr | In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice |
| title_full_unstemmed | In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice |
| title_short | In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice |
| title_sort | in vivo adenine base editing reverts c282y and improves iron metabolism in hemochromatosis mice |
| url | https://doi.org/10.1038/s41467-022-32906-9 |
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