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Transposon-based, targeted ex vivo gene therapy to treat age-related macular degeneration (AMD)

Objetivo

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 and a neuroprotective factor produced by retinal pigment epithelial (RPE) cells result in subretinal neovascularization and retinal pigment cell degeneration. The current treatment by monthly injections of anti-VEGF antibodies is only effective for ~30% of patients. To avoid the severe side effects, high costs and the overall continuing burden on health care associated with monthly antibody injections, inducing a higher level of PEDF expression to inhibit neovascularization would be a viable therapeutic alternative. TargetAMD will subretinally transplant genetically modified, patient-derived, Iris Pigment Epithelial cells that overexpress PEDF to provide a long-lasting cure of AMD. Stable PEDF gene delivery will be based on the non-viral Sleeping Beauty transposon system, which combines the efficacy of viral delivery with the safety of naked DNA plasmids. Academic scientists and SME partners will produce innovative gene delivery technologies, reagents and devices to be translated into a simple and safe gene therapeutic treatment for exudative AMD. Experienced clinicians will perform one clinical trial, comprising isolation and PEDF-transfection of a patient’s pigment epithelial cells and implantation of transfected cells into the patient during a single, 60-minute surgical session. This project will bring a significant enhancement on quality of life to AMD patients, highlight the synergistic power of academic, clinical and industrial cooperation to the scientific arena, and open new markets for novel products for clinical applications of transposon-based gene therapy to industry.

Convocatoria de propuestas

FP7-HEALTH-2012-INNOVATION-1
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Coordinador

UNIVERSITE DE GENEVE
Dirección
Rue du general dufour 24
1211 Geneve
Suiza

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Región
Schweiz/Suisse/Svizzera Région lémanique Genève
Tipo de actividad
Higher or Secondary Education Establishments
Contacto administrativo
Gabriele Thumann (Prof.)
Enlaces
Aportación de la UE
Sin datos

Participantes (14)