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Gene therapy of hemophilia: Hub centres should be haemophilia centres: A joint publication of EAHAD and EHC

9 Mar 2022

Abstract excerpt

Gene therapy has become an increasingly promising, and rapidly developing field, using various innovative techniques to treat, in particular, very rare diseases for which there are no or inadequate treatments. A small number of gene therapies based on adeno-associated viruses (AAV) and lentiviruses have already been approved in the EU and the USA for various hereditary diseases, and several gene therapies are in...

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PMID: 35263819
DOI: 10.1111/hae.14546

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