Biography & Research:
Neurodegenerative diseases are an important problem considering the aging population and the paucity of efficacious treatments. Klein’s lab uses vector gene transfer to study underlying disease processes and to develop novel therapies. Gene transfer to the CNS using adeno-associated virus (AAV) vectors has become an indispensable approach for basic and clinical neuroscience. Klein was one of the early investigators in this space, first overcoming the problem of sustained vs. transient expression in the CNS, and has published over 60 papers on AAV gene transfer in the CNS. His work has helped investigators in the field with respect to understanding the efficiency and the targeting of gene transfer using different promoters, different AAV serotypes, and different vector purification methods. Original neuropathology models from Klein’s group include alpha-synuclein for Parkinson’s disease, tau for Alzheimer’s disease and frontotemporal dementia, and TDP-43 and FUS for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia.