Haemophilia gene therapy: experiences and lessons from treated patients.
(2022) Orphanet Journal of Rare Diseases — Vol. 17, n° 1, p. 154 [1-3] (2022)
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- (en) Two decades of basic research and several recent clinical trials have turned the long-awaited hope of gene therapy for haemophilia into a reality. The principle is to endow liver cells with the ability to produce clotting factor VIII (FVIII) or IX (FIX), whose genetically induced defect in synthesis characterises haemophilia A and B respectively. The aim is to induce sufficient endogenous production of these clotting factors in the long term, thereby ensuring that no haemorrhages occur, particularly in the joints. [...]
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Hermans, C. (2022). Haemophilia gene therapy: experiences and lessons from treated patients. Orphanet Journal of Rare Diseases, 17(1), 154 [1-3]. https://doi.org/10.1186/s13023-022-02313-w (Original work published 2022)