中枢神经系统疾病的基因治疗

基因治疗正在成为常规方法难以治愈的中枢神经系统疾病的新的治疗手段。本文概要介绍了中枢神经系统疾病基因治疗的方法和途径;中枢神经系统疾病基因治疗的病毒载体以及目前中枢神经系统疾病基因治疗研究的状况。

The gene therapy of central nervous system disorders

Gene therapy is a potential and new methods of treating many neurological diseases that were considered refractory to current conventional treatments. By injection of viral vectors or transplantation of genetically engineered cells into specific sites of the brain, the development of technologies for delivery of foreign genes into brain opened the field to promising treatments for CNS disorders. But the development of gene therapy for neurological diseases represents a more complex problem than that for other systemic disorders. Incomplete characterization of target organs (brain and spinal cord) and additional challenges, such as post mitotic cells, heterogeneity of cell types and circuits, and limited access, all contribute to the difficulties. For this reason, this report provides an update outline of gene therapy in CNS and reviews current researches in this filed.