About Ron J Mandel
My research has focused on gene transfer to the nervous system both to treat animal models of neurological disorders and to model neurological disorders. The bulk of my research has focused on Parkinson’s disease. During my second stint at Wallenberg Neuroscience Center, we developed and validated the nigral rAAV-α-synuclein model of Parkinson’s disease. In collaboration with Nicholas Muzyczka, we have continued to use rAAV vectors to study the basic biology of α-synuclein in substantia nigra both by studying various forms of α-synuclein and by knocking down α-synuclein via shRNAs. I have done the most work on rAAV mediated GDNF delivery for the treatment of PD. More recently, I have branched out to gene therapy for many neurological disorders. Currently, I am using rAAV delivered DREADDs to study the basal ganglia circuits involved in repetitive behaviors which may be related to autism.