Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
Progressive familial intrahepatic cholestasis type 3 is a disease caused by mutations in the gene encoding the multidrug resistance protein 3, and has limited treatment options. Here they show that adeno-associated virus mediated gene therapy prevents disease progression in a mouse disease model.
| Main Authors: | , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Publishing Group
2019-12-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-019-13614-3 |