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CC BY V4.0, Open Access
Journal article
Antisense oligonucleotides and their applications in rare neurological diseases
Frontiers in neuroscience, Vol.18, 1414658
2024
Abstract
Rare diseases affect almost 500 million people globally, predominantly impacting children and often leading to significantly impaired quality of life and high treatment costs. While significant contributions have been made to develop effective treatments for those with rare diseases, more rapid drug discovery strategies are needed. Therapeutic antisense oligonucleotides can modulate target gene expression with high specificity through various mechanisms determined by base sequences and chemical modifications; and have shown efficacy in clinical trials for a few rare neurological conditions. Therefore, this review will focus on the applications of antisense oligonucleotides, in particular splice-switching antisense oligomers as promising therapeutics for rare neurological diseases, with key examples of Duchenne muscular dystrophy and spinal muscular atrophy. Challenges and future perspectives in developing antisense therapeutics for rare conditions including target discovery, antisense chemical modifications, animal models for therapeutic validations, and clinical trial designs will also be briefly discussed.
Details
- Title
- Antisense oligonucleotides and their applications in rare neurological diseases
- Authors/Creators
- Simon McDowallMay Aung-HtutSteve WiltonDunhui Li
- Publication Details
- Frontiers in neuroscience, Vol.18, 1414658
- Publisher
- Frontiers Media S.A.
- Number of pages
- 13
- Grant note
The author(s) declare that no financial support was received for the research, authorship, and/or publication of the article.
- Identifiers
- 991005705768607891
- Copyright
- © 2024 McDowall et al.
- Murdoch Affiliation
- Centre for Molecular Medicine and Innovative Therapeutics
- Language
- English
- Resource Type
- Journal article
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- Collaboration types
- Domestic collaboration
- Citation topics
- 2 Chemistry
- 2.170 Nucleic Acids Chemistry
- 2.170.988 Oligonucleotide Modifications
- Web Of Science research areas
- Neurosciences
- ESI research areas
- Neuroscience & Behavior