Inherited retinal degenerations constitute a large and heterogeneous group of untreatable blinding conditions affecting 1 in 2000 people worldwide. They are caused by mutations in one of more than 250 genes mainly expressed in photoreceptor cells, as well as in the retinal pigment epithelium. Retinitis Pigmentosa (RP) and Stargardt disease (STGD1) are amongst the most frequent and severe forms of such retinal degenerative diseases. The identification over the last three decades of genes implicated in retinal degeneration has laid the ground for the development of gene therapies to treat these types of diseases.

The Gene Therapy for Retinal Diseases team has accumulated a wealth of experience in translational research into gene therapies for inherited retinal dystrophies. This includes the preclinical proof-of-concept for retinal gene therapy in canine models of RPE65 and PDE6b deficiencies, leading to Phase I/II clinical trials for these diseases.

The group is currently focusing on adapting the new developments in genome editing to treat Stargardt disease, and on the generation of new animal models of genetic retinal degenerations that more faithfully mimic the human condition.


Team members