Antisense technologies have a future fighting neurodegenerative diseases

Abstract

Our growing understanding of the role that unfavorable patterns of gene expression play in the etiology of neurodegenerative disease emphasizes the need for strategies to selectively block the biosynthesis of harmful proteins in the brain. Antisense technologies are ideally suited to this purpose. Tailor-designed to target specific RNA, antisense oligonucleotides and ribozymes offer tools to suppress the production of proteins mediating neurodegeneration. Although technical limitations must still be overcome, the antisense approach represents a novel and exciting strategy for intervention in diseases of the central nervous system.