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Haemophilia gene therapy: progress and obstacles

Presented By
Prof. Amit Nathwani, University College London, United Kingdom
ISTH 2020
Review study
The future of gene therapy for haemophilia patients looks bright. Prof. Amit Nathwani (University College London, United Kingdom) presented a review of promising gene therapy trial results in haemophilia B and A [1]. Among vectors of viral origin, and as far as is known to date, adeno-associated virus (AAV) vectors have the best safety profile. “AAV vectors can integrate into the host DNA but at frequencies that are quite low,” Prof. Nathwani explained. However, this integration has only been shown in animal studies until today. A single administration of AAV vectors leads to stable long-term expression of transgenic protein. Haemophilia B: fewer factor concentrates necessary AAV comes in a variety of different serotypes (e.g. AAV2, AAV5, AAV8, AAV9) and selection of the most suitable capsid is required. For example, Prof. Nathwani and colleagues selected an...

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