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Malignancy and gene therapy in hemophilia

Despite the misconception that adeno-associated virus (AAV) gene therapy vectors are nonintegrating, they can integrate into the host genome at a low but nonnegligible frequency, posing a theoretical risk of tumorigenesis. While AAV integration can trigger hepatocellular carcinoma in mice, no such a...

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Hovedforfatter: Radoslaw Kaczmarek
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2026-01-01
Serier:Research and Practice in Thrombosis and Haemostasis
Fag:
AAV
AAV oncogenicity
AAV tumorigenicity
gene therapy
hemophilia
vector integration
Online adgang:http://www.sciencedirect.com/science/article/pii/S2475037925006077
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