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Updated: Jan 24, 2026

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Published on: October 29, 2019

Antisense oligonucleotides: A primer.

Daniel R Scoles¹, Eric V Minikel¹, Stefan M Pulst¹

¹Department of Neurology (D.R.S., S.M.P.), University of Utah, Salt Lake City, UT; and Center for the Science of Therapeutics (E.V.M.), Broad Institute of MIT and Harvard, Cambridge, MA.

Neurology. Genetics

|May 24, 2019

Summary

This summary is machine-generated.

Antisense oligonucleotides (ASOs) show promise for treating neurodegenerative diseases. Current research and industry focus highlight ASOs as a key future therapeutic strategy for conditions like Alzheimer's and Parkinson's.

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Area of Science:

Neurology
Genetics
Pharmacology

Background:

Limited disease-modifying treatments exist for neurodegenerative diseases.
Successful development of antisense oligonucleotides (ASOs) for spinal muscular atrophy and Duchenne muscular dystrophy indicates therapeutic potential.
Growing pipelines and industry interest in ASO therapies for various neurodegenerative conditions are evident.

Purpose of the Study:

To review the different types of antisense oligonucleotides (ASOs).
To describe the therapeutic applications of ASOs.
To outline current efforts in developing novel ASO therapies for neurodegenerative diseases.

Main Methods:

Literature review of existing and developing antisense oligonucleotide (ASO) therapies.
Analysis of ASO therapeutic advantages over small molecule approaches.
Examination of ASO development pipelines for neurodegenerative diseases.

Main Results:

Antisense oligonucleotide (ASO) therapeutics have demonstrated success in specific genetic disorders.
Numerous neurodegenerative diseases, including spinocerebellar ataxias, Huntington's, Alzheimer's, ALS, and Parkinson's, are targets for ASO therapy development.
The target sequence acquisition for ASOs offers a direct route to therapeutic oligonucleotide design.

Conclusions:

Antisense oligonucleotides (ASOs) represent a promising therapeutic modality for neurodegenerative diseases.
The development of ASO therapies is expanding, driven by successes and a strong industry focus.
ASOs are poised to become a significant part of the future therapeutic toolkit for a range of neurological disorders.