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In Parkinson’s disease, parkinsonism occurs due to the loss of dopaminergic neurons of the substantia nigra. Existing treatments can enhance dopaminergic activity in the brain, but cause adverse effects due to the non-targeted, non-physiologic dopamine delivery, so there is interest in developing regenerative therapies to restore dopaminergic tone in the striatum in a targeted, physiologic manner. Experimental approaches include using viral vectors to deliver genes encoding growth factors or enzymes involved in dopamine synthesis, or to target nucleic acids and gene expression. A number of cell types have been considered potential sources of cell-based therapies for PD and have been trialled in humans and animals, but all have been limited by either poor efficacy, poor graft survival, or logistical barriers. However, stem cells offer a renewable source of dopaminergic cells and hold great promise as potential regenerative treatments, and human trials have begun. Although these treatments remain experimental, some are entering clinical trials and there is hope that they will become available for clinical use in the future.
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