Gene therapy approaches in the non-human primate model of Parkinson's disease.

摘要: The field of gene therapy has recently witnessed a number major conceptual changes. Besides the traditional thinking that comprises use viral vectors for delivery given therapeutic gene, original approaches have been envisaged, focused on using carrying genes to further modify basal ganglia circuits interest. It is expected these will ultimately induce potential being sustained by gene-induced changes in brain circuits. Among others, at present, it technically feasible (1) achieve controlled release neurotrophic factors, (2) conduct either transient or permanent silencing any circuit interest, (3) perform an vivo cellular reprogramming promoting conversion resident cells into dopaminergic-like neurons, and (4) improving levodopa efficacy over time targeting aromatic l-amino acid decarboxylase. Furthermore, extensive research efforts based are currently ongoing attempt better replicate dopaminergic neurodegeneration phenomena inherent progressive intraneuronal aggregation alpha-synuclein. Finally, incoming strategies soon emerge horizon, underlying goal alpha-synuclein clearance, such as, instance, initiatives increasing activity glucocerebrosidase. To provide adequate proof-of-concept safety push forward true translational different types therapies before entering clinical trials, non-human primate models undoubtedly plays instrumental role.