View abstract

This abstract is assigned to session BVVB-OBPC: Belgische Vereniging ter Voorkoming van Blindheid/Organisation Belge pour la Prévention de la Cécité

Title	Gene-therapy for retinal dystrophies
Abstract Nr.	356

Purpose	To provide an update on gene therapy for retinal dystrophies.
Methods	An overview of the results of gene therapy trials in humans and animals will be presented.
Results	Gene 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.
Conclusion	Gene 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.

Author 1

Last name	LEROY
Initials	BP
Department	Dept of Ophthalmology & Ctr for Medical Genetics, Ghent University Hospital
City	Ghent