Updates on C9orf72 and TDP-43 Research from Dr Jeffrey Rothstein

Dr Jeffrey Rothstein is a Professor, Department of Neurology and Neuroscience and Director for the Brain Science institute a neurologist/neurobiologist with a major commitment to investigations of the biology underlying neurodegenerative disease, with a specific focus on amyotrophic lateral sclerosis. His research focuses, of several pathways of cellular dysfunction including a major focus on the biology of the nuclear pore, nucleoporins and nuclear transport in ALS, Huntingtons disease and dementias.
Recent work in his lab is of direct import to the C9orf72 repeat expansion , including a new approach to ASOs for C9 by directly targeting the anti-sense RNA which was not part of the two unsuccessful C9 aso trials previously.