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Haemophilia gene therapy: experiences and lessons from treated patients.
- Source :
- Orphanet journal of rare diseases, Vol. 17, no. 1, p. 154 [1-3] (2022)
- Publication Year :
- 2022
-
Abstract
- 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. [...]
Details
- Database :
- OAIster
- Journal :
- Orphanet journal of rare diseases, Vol. 17, no. 1, p. 154 [1-3] (2022)
- Notes :
- English
- Publication Type :
- Electronic Resource
- Accession number :
- edsoai.on1372955158
- Document Type :
- Electronic Resource