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 |
| Summary: | 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 FVIII protein. At present, the main treatment option relies on the injection of expensive clotting-factor concentrates to restore functional levels of the FVIII. Dong-Wook Kim and colleagues at Yonsei University in Seoul, South Korea, have used genome editing techniques to insert a corrected version of the FVIII gene into stem cells derived from a patient with severe hemophilia A. When these cells differentiated into the cells lining blood vessels they were able to produce and secrete active FVIII protein. This approach offers the attractive possibility of correcting all hemophilia-causing FVIII mutations. |
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| ISSN: | 1226-3613 2092-6413 |