Restoration of FVIII expression by targeted gene insertion in the FVIII locus in hemophilia A patient-derived iPSCs
Hemophilia: One gene therapy fits all A strategy to restore the expression of the gene encoding blood clotting factor VIII (FVIII) offers new hope to patients with hemophilia A. Hemophilia A is a rare bleeding disorder caused by a variety of mutations in the FVIII gene which affect the function of F...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Publishing Group
2019-04-01
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| Series: | Experimental and Molecular Medicine |
| Online Access: | https://doi.org/10.1038/s12276-019-0243-1 |