RD-CURE

Bringing Gene Supplementation Therapy for PDE6 Associated Retinopathies into Clinical Practice

01.10.2014 bis 30.09.2016

This project part (WP2) of the RD-CURE consortium will generate a new, human homologous animal model for compound heterozygote Pde6a RP. Detailed characterization of this new model will be used to establish AAV mediated Pde6a gene replacement therapy. In parallel, neuroprotective in vitro and in vivo approaches, with drugs already approved for clinical use, will be tested for their potential to widen the therapeutic window-of-opportunity for gene therapy.
WP2 will also provide clinically relevant biomarkers for both rod and cone system rescue that will serve to validate gene therapy treatments, initially in animal models and later (in WP5) in human patients.

Ÿ- Generation and characterization of a Pde6a (R562W/V685M) compound heterozygote mouse model homologous to human Retinitis Pigmentosa
Ÿ- Development of reliable biomarkers for rod and cone system rescue
Ÿ- Validation of gene therapy and neuroprotective strategies in the Pde6a mouse model