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Article

The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art

2024-09-04

Abstract excerpt

Despite major advances in prophylaxis, no repeated dosing regimen with currently employed extended-half-life or non-factor products replaces the advantages of a long-term cure in persons with severe congenital hemophilia A and B (HA, HB). They indeed live with the risk of breakthrough bleedings, and treatment is still invasive, both physically and psychologically. Early studies showed that adeno-associated virus-b...

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Identifiers and source

Literature Corpus work
16d241ff-be7b-5cf8-a400-986bc1aa4802
DOI
10.4081/btvb.2024.144
Open publication

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