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Haemophilia gene therapy: experiences and lessons from treated patients.

Authors :
UCL - SSS/IREC/CARD - Pôle de recherche cardiovasculaire
UCL - (SLuc) Centre de malformations vasculaires congénitales
UCL - (SLuc) Service d'hématologie
Hermans, Cédric
UCL - SSS/IREC/CARD - Pôle de recherche cardiovasculaire
UCL - (SLuc) Centre de malformations vasculaires congénitales
UCL - (SLuc) Service d'hématologie
Hermans, Cédric
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