Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates
Liver gene therapy with adeno-associated viral (AAV) vectors delivering clotting factor transgenes into hepatocytes has shown multiyear therapeutic benefit in adults with hemophilia. However, the mostly episomal nature of AAV vectors challenges their application to young pediatric patients. We devel...
Main Authors: | , , , , , , , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Research
2022
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Online Access: | View Fulltext in Publisher |