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Gene therapy for haemophilia A and B, from basic principles to clinical implementation: An illustrated review

18 Dec 2023

Abstract excerpt

INTRODUCTION: With recent approval of the first two gene therapies for haemophilia A and B, educational materials about AAV-based gene therapy are needed by the haemophilia community for a better understanding of this novel therapeutic approach and helping healthcare providers and patients making personalized choices amongst an increasing array of therapeutic options. AIM: To provide a comprehensive summary of...

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PMID: 38111029
DOI: 10.1111/hae.14907

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