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Related Experiment Video

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Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
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Therapeutic antisense oligonucleotides for movement disorders.

Epaminondas Doxakis1

  • 1Center of Basic Research, Biomedical Research Foundation, Academy of Athens, Athens, Greece.

Medicinal Research Reviews
|July 14, 2020
PubMed
Summary

Antisense oligonucleotide (ASO) therapies offer new hope for movement disorders by targeting RNA. This review explores ASO technology, its application in neurological conditions, and future potential.

Keywords:
Huntington′s diseaseParkinson′s diseaseantisense oligonucleotidemovement disorderspharmacokineticsspinocerebellar ataxiastherapytoxicology

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Area of Science:

  • Neurology
  • Molecular Biology
  • Genetics

Background:

  • Movement disorders, including akinetic and hyperkinetic syndromes, lack effective therapies.
  • Many movement disorders have a genetic basis, making them suitable for targeted genetic therapies.
  • Antisense oligonucleotide (ASO) therapy represents a promising new approach for neurological conditions.

Purpose of the Study:

  • To review the current state of antisense oligonucleotide (ASO) therapies for movement disorders.
  • To discuss the mechanisms, chemical modifications, delivery, and toxicities of ASOs.
  • To summarize preclinical and clinical trial data for ASO treatments in Parkinson's disease, Huntington's disease, and ataxias.

Main Methods:

  • Review of existing literature on ASO technology and its application in neurological disorders.
  • Analysis of ASO chemical modifications, RNA binding mechanisms, and delivery systems.
  • Examination of preclinical and clinical trial outcomes for ASO therapies in specific movement disorders.

Main Results:

  • ASOs can modulate gene expression and splicing defects, offering a versatile therapeutic strategy.
  • Significant progress has been made in developing ASO therapies since their initial approval in 2016.
  • Preclinical and clinical studies show potential for ASOs in treating Parkinson's, Huntington's, and various ataxias.

Conclusions:

  • ASO therapies hold significant promise for treating a range of movement disorders.
  • Further research and attention to specific challenges are needed to fully realize the potential of ASOs.
  • The development of ASO therapies marks a paradigm shift in the treatment of genetically influenced neurological conditions.