CHAPTER 8 – Gene Therapy for Parkinson's Disease

摘要: Parkinson's disease (PD) is a debilitating, neurodegenerative disorder resulting from loss of dopaminergic neurons in the substantia nigra pars compacta leading to distressing motor symptoms. The current standard pharmacological treatment for PD direct replacement dopamine by with its precursor, levodopa (L-dopa). However, this does not significantly alter progression and becomes less efficacious over time. As result, significant amount research last decade has been aimed at development novel therapeutics such as gene therapy. multi-factorial aetiology sporadic expanding list genetic mutations linked familial forms means that it unlikely any single will cure disease. least three distinct transfer strategies are currently being pursued — genes enzymes involved production, growth factors cell survival regeneration, use reset neuronal circuitry switching cellular phenotype. pre-clinical data merits each these discussed along other potential therapy targets manipulation ubiquitin–proteasome system anti-apoptotic factors. Also highlighted remaining concerns may impede technology clinic treatment.