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Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
Published on: October 29, 2019
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Nucleic acid therapeutics in neurodevelopmental disease
Audrey M Winkelsas1, Kenneth H Fischbeck2
1Neurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, United States; Nuffield Department of Medicine, University of Oxford, Oxford, United Kingdom.
Current Opinion in Genetics & Development
|July 6, 2020
Summary
Nucleic acid therapeutics offer targeted gene therapies for neurological diseases. This review explores approved and developing treatments like antisense oligonucleotides (ASOs) and RNA interference (siRNA) for neurodevelopmental disorders.
Area of Science:
- Biotechnology
- Genetics
- Neuroscience
Background:
- Nucleic acid therapeutics enable precise targeting of disease-causing genes.
- These therapies can modulate gene expression to combat genetic disorders.
- Approved treatments include antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and gene-replacement therapies.
Purpose of the Study:
- To review the applications and limitations of nucleic acid therapeutics.
- To highlight clinical and preclinical examples for neurodevelopmental diseases.
- To discuss challenges in neuronal target engagement and phenotype amelioration.
Main Methods:
- Literature review of nucleic acid therapeutic modalities.
- Analysis of FDA-approved and investigational treatments.
- Focus on applications in neurodevelopmental disorders.
Main Results:
- Several nucleic acid therapeutic classes are approved and in development.
- Challenges exist in delivering these therapies to neurons effectively.
- Demonstrated potential for treating neurological conditions.
Conclusions:
- Nucleic acid therapeutics represent a promising approach for neurodevelopmental diseases.
- Further research is needed to optimize delivery and efficacy in the nervous system.
- These modalities offer new avenues for genetic medicine in neurology.
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