Antisense Oligonucleotides for Amyotrophic Lateral Sclerosis

Sean E. Smith; Timothy M. Miller

doi:10.1007/978-3-031-89307-0_11

Antisense Oligonucleotides for Amyotrophic Lateral Sclerosis

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

Abstract

Antisense oligonucleotides (ASOs) 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 cerebrospinal fluid (CSF) to achieve widespread distribution throughout the brain and spinal cord. ASOs have been used to target genes associated with amyotrophic lateral sclerosis (ALS), with recent FDA approval for Tofersen for SOD1-related ALS. ASO approaches are also in clinical trial for additional ALS-related genes, including FUS, C9orf72, Ataxin-2, Stathmin-2, and others. ASOs 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 Science+Business Media
Pages	185-200
Number of pages	16
ISBN (Electronic)	9783031893070
ISBN (Print)	9783031893063
DOIs	https://doi.org/10.1007/978-3-031-89307-0_11
State	Published - Jan 1 2025

Keywords

Amyotrophic lateral sclerosis
C9orf72
miRNA
mRNA
SOD1

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10.1007/978-3-031-89307-0_11

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AB - Antisense oligonucleotides (ASOs) 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 cerebrospinal fluid (CSF) to achieve widespread distribution throughout the brain and spinal cord. ASOs have been used to target genes associated with amyotrophic lateral sclerosis (ALS), with recent FDA approval for Tofersen for SOD1-related ALS. ASO approaches are also in clinical trial for additional ALS-related genes, including FUS, C9orf72, Ataxin-2, Stathmin-2, and others. ASOs are a promising therapeutic approach for ALS.

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KW - mRNA

KW - SOD1

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ER -

Antisense Oligonucleotides for Amyotrophic Lateral Sclerosis

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Keywords

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