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Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates
- Source :
- Nature Communications, Vol 13, Iss 1, Pp 1-14 (2022)
- Publication Year :
- 2022
- Publisher :
- Nature Portfolio, 2022.
-
Abstract
- “Lentiviral gene therapy to the liver establishes stable long-term normal to supra-normal coagulation factor VIII activity in mouse models of hemophilia A and in non-human primates, representing a potential new treatment option for people with hemophilia A.”.
- Subjects :
- Science
Subjects
Details
- Language :
- English
- ISSN :
- 20411723
- Volume :
- 13
- Issue :
- 1
- Database :
- Directory of Open Access Journals
- Journal :
- Nature Communications
- Publication Type :
- Academic Journal
- Accession number :
- edsdoj.9b97a0d651b468fbb352c8d225f07b9
- Document Type :
- article
- Full Text :
- https://doi.org/10.1038/s41467-022-30102-3