In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice

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...

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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Nature Portfolio 2022-09-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-022-32906-9

Internet

https://doi.org/10.1038/s41467-022-32906-9

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