Antisense strategies for the treatment of neurological disease

Abstract

Antisense approaches are increasingly being used as a tool to elucidate biological mechanisms. The use of antisense as a therapeutic strategy has been hampered by a number of problems, including stability, delivery and non-specific toxicity. In this paper the patent literature from 1997 - 2000 is reviewed, in which antisense is claimed for the treatment of neurological disorders, including neurodegenerative diseases, stroke, multiple sclerosis, trauma and brain tumour. Although numerous claims are made, in most cases there is very little supporting biological data, particularly with respect to disease models. While antisense strategies offer great promise in terms of the potential to target pathogenic genes in a selective fashion, considerable work remains to demonstrate efficacy in vivo and to ensure adequate delivery without toxicity. Antisense may also prove useful as a tool for imaging gene expression.

• Alzheimer’s disease
• antisense
• apoptosis
• imaging
• multiple sclerosis
• neoplasm
• neurodegeneration
• Parkinson’s disease
• stroke
• traumatic brain injury