2 projets européens trouvés
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TERMINÉ
Clinical trial of gene therapy for MPS VI - a severe lysosomal storage disorder (MEUSIX)
Date du début: 1 déc. 2012, Date de fin: 30 nov. 2017,
"Mucopolysaccharidosis VI (MPS VI, or Maroteaux-Lamy syndrome; OMIM #253200) is a rare lysosomal storage disease caused by deficient activity of arylsulfatase B (ARSB). MPS VI is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement. Thus, systemic therapies targeting peripheral organs have the pot ...
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Participants partenaires
TERMINÉ
Transposon-based, targeted ex vivo gene therapy to treat age-related macular degeneration (AMD) (TARGETAMD)
Date du début: 1 nov. 2012, Date de fin: 31 oct. 2016,
Age-related Macular Degeneration (AMD), a neurodegenerative disease of the retina, is a major cause of blindness in elderly people. Due to the aging population, AMD has been referred to as a “time bomb” in society. In the exudative form of AMD, high levels of vascular endothelial cell growth factor (VEGF) and low levels of pigment-epithelial derived factor (PEDF), an inhibitor of vascularization a ...
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