Gene transfer in hemophilia A: not cogent yet
The phase 3 clinical study of gene transfer in hemophilia A using the BioMarin vector AAV5-hFVIII-SQ (also identified as valoctocogene roxaparvovec) recently reported the results obtained in as many as 134 adult men with severe hemophilia A, so that it is at the moment the largest gene transfer stu...
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| Format: | Article |
| Language: | English |
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PAGEPress Publications
2022-04-01
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| Series: | Bleeding, Thrombosis and Vascular Biology |
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| Online Access: | https://btvb.org/btvb/article/view/32 |