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Article

Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A

18 Jul 2024

Abstract excerpt

ABSTRACT: Following systemically administered adeno-associated virus gene therapy, vector particles are widely distributed, raising concerns about horizontal or germline vector transmission. Characterization of biodistribution and kinetics of vector DNA in body fluids can address these concerns and provide insights into vector behavior in accessible samples. We investigated biodistribution and vector shedding...

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Share to Blood Coagulation and Thrombosis Mechanisms
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 39024543
DOI: 10.1182/bloodadvances.2024013150

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