Prof. Offen's lab focuses on developing cell-based and gene-based therapies for neurodegenerative diseases .
Advanced methods are used, such as CRISPR/Cas9 for in vivo gene modification and multiple platforms for the delivery of therapeutics into the CNS, including mesenchymal stem cells, exosomes and peptides. The effect of gene modification on cognition and behaviour, as well as on disease-related biochemical and histological features are evaluated in cultures and animal models. We used novel techniques to track the migration and homing patterns of intranasally administrated exosomes derived from mesenchymal stem cells in different murine models including stroke, autism, Parkinson’s disease, Alzheimer’s disease and spinal cord injury. We demonstrated that intranasal administration significantly improves motor and behavioral symptoms. We hope that the marked beneficial effects of the exosomes in mice models will translate to a novel, non -invasive, therapeutic strategy in neurological disorders.
Our studies leads to several patents, some of which served as the basis for start-up companies. Among them: "Brainstorm Cell Therapeutics”.
Brainstorm is developing a pioneering technology, currently in a phase III trial in the USA with 200 patients, which has proved effective in treating patients suffering from ALS. Furthermore, the discoveries on gene therapy and
exosomes, were licensed to address neuronal indications, including neuropathy
and reducing autism symptoms.