Mar 24, 2015

Antisense oligonucleotides in therapy for neurodegenerative disorders

Advanced Drug Delivery Reviews
Melvin M EversWilleke M C van Roon-Mom

Abstract

Antisense oligonucleotides are synthetic single stranded strings of nucleic acids that bind to RNA and thereby alter or reduce expression of the target RNA. They can not only reduce expression of mutant proteins by breakdown of the targeted transcript, but also restore protein expression or modify proteins through interference with pre-mRNA splicing. There has been a recent revival of interest in the use of antisense oligonucleotides to treat several neurodegenerative disorders using different approaches to prevent disease onset or halt disease progression and the first clinical trials for spinal muscular atrophy and amyotrophic lateral sclerosis showing promising results. For these trials, intrathecal delivery is being used but direct infusion into the brain ventricles and several methods of passing the blood brain barrier after peripheral administration are also under investigation.

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Mentioned in this Paper

Nested Transcripts
Blood - Brain Barrier Anatomy
Pre-mRNA Splicing
Drug Delivery Systems
Spinal Muscular Atrophy
Structure-Activity Relationship
Brain
Degenerative Diseases, Spinal Cord
Neurodegenerative Disorders Pathway
Revival

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