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Deze abstract is toegekend aan sessie BVVB-OBPC: Belgische Vereniging ter Voorkoming van Blindheid/Organisation Belge pour la Prévention de la Cécité
TitelGene-therapy for retinal dystrophies
Abstract Nr.356
DoelTo provide an update on gene therapy for retinal dystrophies.
MethodesAn overview of the results of gene therapy trials in humans and animals will be presented.
ResultatenGene therapy has been successful in the treatment of Leber congenital amaurosis in a number of independent human trials. In addition, numerous trials in animal models of retinal dystrophies have proven efficacy of this type of treatment.
ConclusieGene therapy provides an exciting opportunity for a select group of retinal dystrophies. However, the enormous effort to prepare patients for this type of therapy by careful genotyping and phenotyping represents a considerable challenge.
Auteur 1
NaamLEROY
InitialenBP
InstituutDept of Ophthalmology & Ctr for Medical Genetics, Ghent University Hospital
StadGhent
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