Gene therapeutic strategies for neurodegenerative diseases

Abstract

Gene transfer into the central nervous system by ex vivo or in vivo techniques is a rapidly emerging field in neuroscience. Potential applications of gene therapy for the nervous system include not only congenital single gene disorders, but also brain tumors and acquired chronic diseases. Considerable progress has been made in the understanding of neurodegenerative diseases such as Parkinson's and Alzheimer's disease. As a result, gene therapy for Parkinson's, and possibly Alzheimer's disease could be regarded as a realistic alternative to the limited treatment options currently available. In this review, we highlight the most important developments in gene transfer techniques as well as the newest insights in the mechanisms of some neurodegenerative disorders and put these into the perspective of gene therapeutic strategies for the central nervous system.