Wrangling RNA: Antisense oligonucleotides for neurological disorders
Abstract
Effective treatment of spinal muscular atrophy with antisense oligonucleotide therapy opens the door to treating other neurological disorders with this approach.
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ASOs do not readily cross the blood-brain barrier, which remains a substantial obstacle for their clinical application in treating CNS disorders.
“However, the fact that ASOs do not readily cross the BBB remains one of the substantial obstacles for their clinical application in treating CNS disorders (28, 29).”
From Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection (2020) · cites this paper as [29]
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- Authors
- Talbot, K., & Wood, M. J. A.
- Affiliations
- University of Oxford (GB)
- Corresponding author
- Talbot, Kevin
- Venue
- Science Translational Medicine
- Published
- 2019-09-25
- Volume
- 11
- Issue
- 511
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- OpenAlex
- W2975845651
- Cited by
- 14
- References
- 10
- Citations by year
- 2025: 1 · 2024: 1 · 2023: 2 · 2022: 3 · 2021: 4 · 2020: 3
- FWCI
- 1.38
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Keywords
- antisense oligonucleotides, spinal muscular atrophy, neurological disorders, RNA-targeted therapeutics, gene silencing
- MeSH terms
- Animals, Humans, Nerve Degeneration, Nervous System Diseases, RNA, Oligonucleotides, Antisense
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