Source 1primary paper2025MED
Toolkit/antisense oligonucleotide
antisense oligonucleotide
Also known as: ASO, ASOs
Taxonomy: Mechanism Branch / Component. Workflows sit above the mechanism and technique branches rather than replacing them.
Summary
ASOs are under development to reduce expression of pathogenic proteins such as tau, α-synuclein, and mutant huntingtin.
Usefulness & Problems
Why this is useful
ASOs are described as being developed to reduce expression of pathogenic proteins including tau, α-synuclein, and mutant huntingtin.; reducing expression of pathogenic proteins in neurodegenerative disease; An antisense oligonucleotide is a named nucleic-acid intervention modality included in the review's abbreviation list. In the abstract context, it is relevant because toxic repeat RNA is proposed as a contributor to C9orf72 ALS-FTD pathogenesis.; candidate therapeutic modality context in C9orf72-associated disease discussions
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ASOs are described as being developed to reduce expression of pathogenic proteins including tau, α-synuclein, and mutant huntingtin.
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reducing expression of pathogenic proteins in neurodegenerative disease
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An antisense oligonucleotide is a named nucleic-acid intervention modality included in the review's abbreviation list. In the abstract context, it is relevant because toxic repeat RNA is proposed as a contributor to C9orf72 ALS-FTD pathogenesis.
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candidate therapeutic modality context in C9orf72-associated disease discussions
Problem solved
It addresses pathogenic protein expression in neurodegenerative disorders.; lowering expression of tau, α-synuclein, and mutant huntingtin; It is a plausible RNA-targeting intervention class for diseases involving toxic repeat RNA, which the abstract identifies as a proposed pathogenic factor.; the abstract links disease pathogenesis to toxic repeat RNA, making ASO a directly named modality relevant to RNA-targeting intervention context
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It addresses pathogenic protein expression in neurodegenerative disorders.
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lowering expression of tau, α-synuclein, and mutant huntingtin
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It is a plausible RNA-targeting intervention class for diseases involving toxic repeat RNA, which the abstract identifies as a proposed pathogenic factor.
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the abstract links disease pathogenesis to toxic repeat RNA, making ASO a directly named modality relevant to RNA-targeting intervention context
Problem links
lowering expression of tau, α-synuclein, and mutant huntingtin
LiteratureIt addresses pathogenic protein expression in neurodegenerative disorders.
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It addresses pathogenic protein expression in neurodegenerative disorders.
the abstract links disease pathogenesis to toxic repeat RNA, making ASO a directly named modality relevant to RNA-targeting intervention context
LiteratureIt is a plausible RNA-targeting intervention class for diseases involving toxic repeat RNA, which the abstract identifies as a proposed pathogenic factor.
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It is a plausible RNA-targeting intervention class for diseases involving toxic repeat RNA, which the abstract identifies as a proposed pathogenic factor.
Taxonomy & Function
Primary hierarchy
Mechanism Branch
Component: A low-level RNA part used inside a larger architecture that realizes a mechanism.
Techniques
No technique tags yet.
Target processes
editingImplementation Constraints
cofactor dependency: cofactor requirement unknownencoding mode: genetically encodedimplementation constraint: context specific validationimplementation constraint: payload burdenoperating role: regulator
Use requires an antisense oligonucleotide strategy directed against a disease-relevant target. The abstract does not provide chemistry, dosing, or delivery details.; requires a targetable pathogenic transcript or protein-expression program; The abstract only supports that this modality exists in scope; it does not describe sequence, chemistry, delivery vehicle, or assay requirements.; requires sequence-specific oligonucleotide design and delivery, but the abstract does not provide implementation details
The abstract does not show that ASOs address all disease mechanisms, including DPR toxicity or reduced C9orf72 expression.; the abstract does not state a specific ASO design, target sequence, or demonstrated outcome
Validation
Cell-freeBacteriaMammalianMouseHumanTherapeuticIndep. Replication
Supporting Sources
Ranked Claims
AAVs and lentiviruses have been used for gene delivery in preclinical and clinical studies.
Adeno-associated viruses (AAVs) and lentiviruses have been used for gene delivery in preclinical and clinical studies
MSC-based paracrine support and transplantation of neurons derived from iPSCs are being evaluated as cellular therapies, particularly in Parkinson's disease and Alzheimer's disease.
Cellular therapies, including mesenchymal stem cell (MSC)-based paracrine support and transplantation of neurons derived from induced pluripotent stem cells (iPSCs), are being evaluated, particularly in PD and AD.
ASOs are under development to reduce expression of pathogenic proteins such as tau, α-synuclein, and mutant huntingtin.
ASOs are under development to reduce expression of pathogenic proteins such as tau, α-synuclein, and mutant huntingtin.
Current and developing therapeutic strategies for neurodegeneration include viral vector-based gene delivery, antisense oligonucleotide and RNA interference methods, stem cell transplantation, and genome editing technologies.
In this review, we describe current and developing therapeutic strategies that include viral vector-based gene delivery, antisense oligonucleotide (ASO) and RNA interference methods, stem cell transplantation, and genome editing technologies.
Approval Evidence
2 sources2 linked approval claimsfirst-pass slug antisense-oligonucleotide
ASOs are under development to reduce expression of pathogenic proteins such as tau, α-synuclein, and mutant huntingtin.
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Abbreviation: ASO: antisense oligonucleotide.
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mechanism statementsupports
ASOs are under development to reduce expression of pathogenic proteins such as tau, α-synuclein, and mutant huntingtin.
ASOs are under development to reduce expression of pathogenic proteins such as tau, α-synuclein, and mutant huntingtin.
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scope statementsupports
Current and developing therapeutic strategies for neurodegeneration include viral vector-based gene delivery, antisense oligonucleotide and RNA interference methods, stem cell transplantation, and genome editing technologies.
In this review, we describe current and developing therapeutic strategies that include viral vector-based gene delivery, antisense oligonucleotide (ASO) and RNA interference methods, stem cell transplantation, and genome editing technologies.
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Comparisons
Source-stated alternatives
The abstract mentions RNA interference as a related expression-lowering approach, and also discusses viral gene delivery, stem cell transplantation, and genome editing.; The abstract contrasts multiple pathogenic contributors, including repeat RNA, DPRs, and reduced C9orf72 expression, but does not name alternative intervention tools.
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The abstract mentions RNA interference as a related expression-lowering approach, and also discusses viral gene delivery, stem cell transplantation, and genome editing.
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The abstract contrasts multiple pathogenic contributors, including repeat RNA, DPRs, and reduced C9orf72 expression, but does not name alternative intervention tools.
Source-backed strengths
explicitly linked to pathogenic protein reduction; explicitly named in the review abbreviation list
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explicitly linked to pathogenic protein reduction
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explicitly named in the review abbreviation list
Compared with stem cell transplantation
The abstract mentions RNA interference as a related expression-lowering approach, and also discusses viral gene delivery, stem cell transplantation, and genome editing.
Shared frame: source-stated alternative in extracted literature
Strengths here: explicitly linked to pathogenic protein reduction; explicitly named in the review abbreviation list.
Relative tradeoffs: the abstract does not state a specific ASO design, target sequence, or demonstrated outcome.
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The abstract mentions RNA interference as a related expression-lowering approach, and also discusses viral gene delivery, stem cell transplantation, and genome editing.
Ranked Citations
- 1.