Gene-based therapy of Parkinson's Disease: Translation from animal model to human clinical trial employing convection enhanced delivery.

摘要: The existing treatment of Parkinson’s disease (PD) is directed towards substituting dopamine loss with either replacement therapy or pharmacological therapies aimed at increasing the synapse level. Emerging viable alternatives include use cell-based and gene-based therapeutics. In this review, we discuss efforts in developing vitro vivo models their translation to human clinical trials for distressing prevalent neurodegenerative disorder. Given mismatch between expectations from preclinical data results pivotal trials, drug delivery has been identified as key emerging area translational research due limitation limited efficacy. chief highlights current topic improved methods therapeutic agents. Convection-enhanced (CED), an advanced infusion technique demonstrated utility ex animal recently adopted PD trials. Several studies suggest that magnetic resonance imaging (MRI)-guided navigation accurately targeting real time monitoring viral vector (rCED) future involving detection gene expression restoration dopaminergic function using pro-drug approach will greatly enhance these treatments.