Project: Treating inherited binding disease with a slow released form of the rod-derived cone viability factor protein

Acronym DrEYE (Reference Number: NEURON-047)
Project Topic The protein rod-derived cone viability factor (RdCVF) is secreted by rod photoreceptors and stimulates the metabolism of the cones by binding a receptor on the cell surface of the cones. In retinitis pigmentosa (RP), the mutation-dependent death of rods results in the reduction of the expression of RdCVF and consequently to the lack of metabolic support from the rods to sustain cone outer segment renewal. This triggers the secondary loss of cone function at the fovea that leads to blindness. Administration of RdCVF is a promising mutation-dependent therapy of RP. Long-term RdCVF therapy can be obtained through gene therapy or through the injection of a sustained protein delivery system, as a safer alternative to the delivery of the protein via viral vectors. We developed an injectable physical blend of hyaluronan and methylcellulose modified with a peptide binding partner of the Src homology 3 (SH3) domain, for controlled release of SH3-RdCVF fusion protein. Tre biological activity and sustained release of this protein were validated in vitro. Our objective is to produce SH3-RdCVF protein, to characterize its biochemical properties and to provide the proof of concept of the therapeutic benefit of its utilization in three models of RP. The mouse model will be used for providing efficacy dose in vivo, and a rat model will test for long-term benefit for cone vision. A novel model of RP in a minipig will be explored as a way toward the translation of this therapeutic approach.
Network NEURON Cofund
Call Call for Proposals for Transnational Research Projects on Sensory Disorders

Project partner

Number Name Role Country
1 Sorbonne Université Coordinator France
2 University of Toronto Partner Canada
3 Ludwig-Maximilians-Universität München Partner Germany
4 Weizmann Institute of Science Partner Israel