Dry AMD - Cellular and Genetic Therapies

The growing incidence of neurodegenerative diseases is based on our increasingly aging society as well as the difficulties in establishing defined therapy regimens. For dry age-related macular degeneration (AMD) and the later stage of geographic atrophy (GA), various treatment options exist that only decelerate the progression of the disease. However, no therapy is currently available that can restore the degenerated retinal pigment epithelium (RPE) and/or photoreceptor cells. Cellular and gene-based approaches aim for the regeneration of the degenerated cells and/or the continuous secretion of cell-protecting agents. The article describes the approaches that are currently being investigated in different clinical trials. These trials are based on the use of cell-based drug delivery systems, stem cells of different origins as well as virus-mediated gene therapy approaches. Finally, we give an overview of ongoing therapeutic developments and present our own research activities, which consist of a combination of pigment epithelial cell transplantation and additive non-viral gene therapy to treat retinal degenerative diseases.