Stem Cell Therapies in Neurodegenerative Diseases

Our group’s goal is to develop new therapeutic cell-based treatment approaches for neurodegenerative diseases by using adult stem cells or pluripotent stem cells such as human embryonic stem cells (hESCs) and induced human pluripotent stem cells (hiPSCs). Our aim is to develop clinically acceptable protocols for neural differentiation and to test them in different animal models as potential therapeutic tools to help treat spinal cord injuries and different types of ataxias. We also apply combinatorial approaches using small molecules in order to increase the success of cell-based therapies.

Hereditary retinal dystrophies are another subject of interest in our laboratory. In particular, we are focusing on creating new human cell models using patient-specific hiPSCs, and especially, retinal pigment epithelial (RPE) cells and optic-cup organoids derived from them. These can then be used to investigate the mechanisms driving these diseases, develop treatments, perform toxicity screens, and model development in cells affected by these diseases.

Using our expertise in obtaining and culturing these cell types, we were able to establish hiPSC cell lines from patients suffering from retinitis pigmentosa. Moreover, we intend to correct the faulty genes in these cells using Crispr/Cas9 technology and to optimize the derivation of RPE and photoreceptors from corrected hiPSCs as a cell source for transplantation therapies in animal models with retinal degeneration diseases. In collaboration with other laboratories, we are developing new tools for maintaining high cell viability while delivering RPE and photoreceptor cells into degenerated retina in small and large animal models.