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...
Full description
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
|
|---|