Gene therapy for Parkinson's disease: a decade of progress supported by posthumous contributions from volunteer subjects
Raymond T.Bartus
摘要：Over the past decade,nine separate gene therapy clinical trials for advanced Parkinson's disease (PD) have been launched and completed,involving the dosing of nearly 12-dozen PD volunteers who incurred significant risks to hopefully reduce symptoms and gain a better life.Each study attempted to directly or indirectly correct or compensate for the dysfunction and death of dopamine neurons (Bartus et al.,2014) that originate in the substantia nigra (located in the brain stem),projecting their axon terminals to the putamen (centrally located within each cerebrum).All told,incalculable hours were spent planning and executing these clinical trials,collectively costing hundreds of millions of dollars.After all is said and done-what has been gained?
机标关键词：clinical trialssubstantia nigragene therapybrain stem
论文发表日期：2015-01-01
在线出版日期：2025-08-15（本平台首次上网日期，不代表文献的发表时间）
页数：3( 1586-1588 )
英文信息