Antisense oligonucleotides for amyotrophic lateral sclerosis

Wade K. Self; Timothy M. Miller

doi:10.1007/978-1-4899-7654-3_6

Antisense oligonucleotides for amyotrophic lateral sclerosis

Research output: Chapter in Book/Report/Conference proceeding › Chapter › peer-review

Abstract

Antisense oligonucleotides (ASO) are short DNA-like chemicals that bind to RNA by Watson–Crick base pairing and modulate function of the RNA. These chemicals do not cross the blood brain barrier, but may be delivered directly to the cerebral spinal fl uid (CSF) to achieve widespread distribution throughout the brain and spinal cord. ASO have been used to target genes associated with familial amyotrophic lateral sclerosis (ALS), such as SOD1 and C9orf72 as well as miRNAs.A Phase I trial for SOD1-targeting ASO showed excellent safety and important pharmacodynamics. ASO are a promising therapeutic approach for ALS.

Original language	English
Title of host publication	Translational Neuroscience
Subtitle of host publication	Fundamental Approaches for Neurological Disorders
Publisher	Springer US
Pages	99-109
Number of pages	11
ISBN (Electronic)	9781489976543
ISBN (Print)	9781489976529
DOIs	https://doi.org/10.1007/978-1-4899-7654-3_6
State	Published - Jan 1 2016

Keywords

ALS
C9orf72
SOD1
mRNA
miRNA

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10.1007/978-1-4899-7654-3_6

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AB - Antisense oligonucleotides (ASO) are short DNA-like chemicals that bind to RNA by Watson–Crick base pairing and modulate function of the RNA. These chemicals do not cross the blood brain barrier, but may be delivered directly to the cerebral spinal fl uid (CSF) to achieve widespread distribution throughout the brain and spinal cord. ASO have been used to target genes associated with familial amyotrophic lateral sclerosis (ALS), such as SOD1 and C9orf72 as well as miRNAs.A Phase I trial for SOD1-targeting ASO showed excellent safety and important pharmacodynamics. ASO are a promising therapeutic approach for ALS.

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KW - miRNA

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Antisense oligonucleotides for amyotrophic lateral sclerosis

Abstract

Keywords

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