Stylianos Michalakis, PhD
Department of Ophthalmology
University Hospital LMU Munich
Inherited retinal degenerations are eye diseases that lead to severe visual impairment and even blindness. In most cases, they are triggered by defects in genes encoding key proteins involved in the visual cascade, which are usually specifically expressed in photoreceptors or retinal pigment epithelial cells. With the development of efficient gene therapy vectors based on non-pathogenic recombinant adeno-associated viruses (AAV), it is now possible to develop potentially curative therapeutic approaches for these previously incurable eye diseases.
In my talk, I will give an introduction to specific therapeutic approaches for gene therapy of achromatopsia and retinititis pigmentosa and discuss their development, clinical application and limitations. I will then discuss possible solutions to some of the limitations.