ReviewRNA biology2018
Oligonucleotide therapeutics in neurodegenerative diseases.
Review in RNA biology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
40 citing papers in PubMed, 74 citations in OpenAlex.
- Contrastive Learning Model for Wearable-Based Ataxia Assessment.IEEE transactions on bio-medical engineering · 2026Article
- Therapy-induced mRNA, rRNA and tRNA methylation alterations confer tolerance phenotype in tumor cells: mechanism and implications.International journal of biological sciences · 2026Review
- CRISPR/Cas9 a genomic engineering technology for treatment in ALS mouse models.Regenerative therapy · 2025Review
- At-home wearables and machine learning capture motor impairment and progression in adult ataxias.Brain : a journal of neurology · 2025Article
- Submovements Derived from Wearable Sensors Capture Ataxia Severity and Differ Across Motor Tasks and Directions of Motion.Cerebellum (London, England) · 2025Article
- Establishment of a humanized SCA2 mouse model carrying a CAA disruption preventing CAG repeat expansion in pathogenic genes.Animal models and experimental medicine · 2025Article
- Quantitative Determination of Click-Reactive Antisense Oligonucleotide and Its Reactivity in Rat Brains by Hybridization LC-MS/MS to Support Pretargeted PET Imaging.Analytical chemistry · 2025Article
- Spinocerebellar Ataxia Type 2: A Review and Personal Perspective.Neurology. Genetics · 2025Review
- Significance of gene therapy in neurodegenerative diseases.Frontiers in neuroscience · 2025Review
- L-arginine in patients with spinocerebellar ataxia type 6: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.EClinicalMedicine · 2024Article
- Structural basis for a highly conserved RNA-mediated enteroviral genome replication.Nucleic acids research · 2024Article
- Article
- Evaluating the efficacy of purchased antisense oligonucleotides to reduce mouse and human tauFrontiers in molecular neuroscience · 2023Article
- Real-life ankle submovements and computer mouse use reflect patient-reported function in adult ataxias.Brain communications · 2023Article
- Recent Updates on the Genetics of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia.Molecular neurobiology · 2022Review
- Partners in crime: Proteins implicated in RNA repeat expansion diseases.Wiley interdisciplinary reviews. RNA · 2022Review
- Consensus Paper: Strengths and Weaknesses of Animal Models of Spinocerebellar Ataxias and Their Clinical Implications.Cerebellum (London, England) · 2022Article
- Radiation enhances the delivery of antisense oligonucleotides and improves chemo-radiation efficacy in brain tumor xenografts.Cancer gene therapy · 2022Article
- Nanocarriers for Delivery of Oligonucleotides to the CNS.International journal of molecular sciences · 2022Review
- Detection of Oculomotor Dysmetria From Mobile Phone Video of the Horizontal Saccades Task Using Signal Processing and Machine Learning Approaches.IEEE access : practical innovations, open solutions · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
Therapeutics that directly target RNAs are promising for a broad spectrum of disorders, including the neurodegenerative diseases. This is exemplified by the FDA approval of Nusinersen, an antisense oligonucleotide (ASO) therapeutic for spinal muscular atrophy (SMA). RNA targeting therapeutics are currently under development for amyotrophic lateral sclerosis (ALS), Huntington's disease (HD), and spinocerebellar ataxias. We have used an ASO approach toward developing a treatment for spinocerebellar ataxia type 2 (SCA2), for targeting the causative gene ATXN2. We demonstrated that reduction of ATXN2 expression in SCA2 mice treated by intracerebroventicular injection (ICV) of ATXN2 ASO delayed motor phenotype onset, improved the expression of several genes demonstrated abnormally reduced by transcriptomic profiling of SCA2 mice, and restored abnormal Purkinje cell firing frequency in acute cerebellar sections. Here we discuss RNA abnormalities in disease and the prospects of targeting neurodegenerative diseases at the level of RNA control using ASOs and other RNA-targeted therapeutics.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.