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Updated: Jan 20, 2026

Gene Therapy in Disease Intervention
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Gene Therapy in Disease Intervention

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Malignancy and gene therapy in hemophilia

Radoslaw Kaczmarek1

  • 1Department of Pediatrics, Herman B Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, Indiana, USA.

Research and Practice in Thrombosis and Haemostasis
|January 19, 2026
PubMed
Summary

Adeno-associated virus (AAV) gene therapy vectors can integrate into the human genome, posing a theoretical cancer risk. Current evidence does not link AAV integration to human cancers, but robust molecular analysis is crucial for definitive conclusions.

Keywords:
AAVAAV oncogenicityAAV tumorigenicitygene therapyhemophiliavector integration

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