Karin Meijboom

Currently I work on gene therapy for diseases of the central nervous system. My main project is on amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. The project is specifically focused on a hexanucleotide repeat mutation found in chromosome 9 open reading frame 72 (C9orf72) that is toxic and causes ALS pathology. I use different strategies to eliminate or silence this toxic repeat, such as gene editing and the use of microRNAs. My other project is on Neurofibromatosis 2 (NF2), a disease that is caused by mutations in the NF2 gene that result in the growth of multiple tumors throughout the central and peripheral nervous systems. For this, I am designing a viral vector that can deliver a functioning copy of the gene