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Updated: Dec 6, 2025
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Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
Published on: October 29, 2019
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Antisense Drugs Make Sense for Neurological Diseases
C Frank Bennett1, Holly B Kordasiewicz1, Don W Cleveland2
1Ionis Pharmaceuticals Inc., Carlsbad, California 92010, USA;
Annual Review of Pharmacology and Toxicology
|October 9, 2020
Summary
Antisense oligonucleotides (ASOs) offer a promising new therapy for neurodegenerative diseases by targeting RNA. This approach is advancing treatments for conditions like Alzheimer's and Parkinson's disease.
Area of Science:
- Neurology
- Genetics
- Pharmacology
Background:
- Genetic basis for inherited neurodegenerative diseases is known, but effective therapies are limited.
- Antisense oligonucleotides (ASOs) represent a novel therapeutic class for neurological disorders.
- ASO mechanism involves RNA degradation or splicing modulation.
Purpose of the Study:
- To review advances in antisense oligonucleotide technology for neurological diseases.
- To highlight the therapeutic potential of ASOs in treating neurodegenerative conditions.
Main Methods:
- Review of current literature on antisense oligonucleotide technology.
- Analysis of ASO applications in preclinical and clinical studies for neurological diseases.
- Examination of ASO delivery methods, including intrathecal injection.
Main Results:
- ASOs demonstrate potential for disease modification in neurodegenerative diseases.
- Intrathecal delivery ensures broad distribution and long-term effects of ASO drugs.
- Nusinersen's approval signifies successful ASO therapeutic application, slowing progression and improving symptoms.
- ASOs are in development for ALS, Huntington's, Alzheimer's, Parkinson's, and Angelman syndrome.
Conclusions:
- Antisense oligonucleotide technology is a promising platform for developing disease-modifying therapies for neurological diseases.
- Further research and clinical development of ASOs are crucial for addressing unmet needs in neurodegenerative disease treatment.
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