Antisense therapies for movement disorders.

Antisense therapies for movement disorders.

Publication date: Jul 08, 2019

Currently, few disease-modifying therapies exist for degenerative movement disorders. Antisense oligonucleotides are small DNA oligonucleotides, usually encompassing ∼20 base pairs, that can potentially target any messenger RNA of interest. Antisense oligonucleotides often contain modifications to the phosphate backbone, the sugar moiety, and the nucleotide base. The development of antisense oligonucleotide therapies spinal muscular atrophy and Duchenne muscular dystrophy suggest potentially wide-ranging therapeutic applications for antisense oligonucleotides in neurology. Successes with these two diseases have heightened interest in academia and the pharmaceutical industry to develop antisense oligonucleotides for several movement disorders, including, spinocerebellar ataxias, Huntington’s disease, and Parkinson’s disease. Compared to small molecules, antisense oligonucleotide-based therapies have an advantage because the target disease gene sequence is the immediate path to identifying the therapeutically effective complementary antisense oligonucleotide. In this review we describe the different types of antisense oligonucleotide chemistries and their potential use for the treatment of human movement disorders. © 2019 International Parkinson and Movement Disorder Society.

Scoles and Pulst, S.M. Antisense therapies for movement disorders. 06569. 2019 Mov Disord.

Concepts Keywords
Antisense Antisense therapy
Antisense Oligonucleotide Sense
Base Pairs Duchenne muscular dystrophy
Duchenne Muscular Dystrophy Muscular dystrophy
Huntington Oligonucleotide
Moiety RNA
Movement Disorders DNA
Neurology Branches of biology
Nucleotide
Oligonucleotides
Parkinson
Pharmaceutical Industry
Phosphate
Spinal Muscular Atrophy
Spinocerebellar Ataxias
Sugar

Semantics

Type Source Name
disease MESH spinal muscular atrophy
disease MESH development
disease MESH movement disorders
disease MESH spinocerebellar ataxias
disease DOID Duchenne muscular dystrophy
disease MESH Duchenne muscular dystrophy
disease DOID spinal muscular atrophy

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