GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice.

EMBO Mol Med · 2024 · PMC11178766 · PMID 38684862

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a decreasing trendno p-value reported

The paper explained Problem The currently approved gene therapy for the inherited bleeding disease hemophilia A is limited by a decreasing trend in the reconstituted coagulation factor VIII (FVIII) activity and its expected duration is in the range of 5–10 years in adults.

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