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Cell-based therapies are promising future approaches to treat Parkinson disease (PD). In this company-funded study, researchers aimed at a new target: the overexcited subthalamic nucleus, which sends excitatory projections to the globus pallidus and substantia nigra pars reticulata. The treatment involves injecting adeno-associated virus vector expressing glutamic acid decarboxylase (AAV-GAD) into these projecting subthalamic neurons, converting them into GABA-producing, inhibitory cells and normalizing downstream motor output signals. In this study, 12 patients received varying doses of AAV-GAD injections into these subthalamic neurons.
The treatment’s safety profile was good and the procedure was well-tolerated, regardless of AAV-GAD dose.…