ReviewGene therapy2011
Using non-coding small RNAs to develop therapies for Huntington's disease.
Review in Gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
What it found
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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
25 citing papers in PubMed, 37 citations in OpenAlex.
- Small interfering RNAs based therapies for intracerebral hemorrhage: challenges and progress in drug delivery systems.Neural regeneration research · 2022Review
- Review
- Recent Overview of the Use of iPSCs Huntington's Disease Modeling and Therapy.International journal of molecular sciences · 2020Review
- Article
- Elimination of huntingtin in the adult mouse leads to progressive behavioral deficits, bilateral thalamic calcification, and altered brain iron homeostasis.PLoS genetics · 2017Article
- Bone scaffolds loaded with siRNA-Semaphorin4d for the treatment of osteoporosis related bone defects.Scientific reports · 2016Article
- N-terminal Huntingtin Knock-In Mice: Implications of Removing the N-terminal Region of Huntingtin for Therapy.PLoS genetics · 2016Article
- Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis.Proceedings of the National Academy of Sciences of the United States of America · 2016Article
- Regulation of mRNA Translation by MID1: A Common Mechanism of Expanded CAG Repeat RNAs.Frontiers in cellular neuroscience · 2016Article
- The epigenetics of aging and neurodegeneration.Progress in neurobiology · 2015Review
- Transcription, epigenetics and ameliorative strategies in Huntington's Disease: a genome-wide perspective.Molecular neurobiology · 2015Review
- Causes and Consequences of MicroRNA Dysregulation in Neurodegenerative Diseases.Molecular neurobiology · 2015Review
- Cell-based therapies for Huntington's disease.Drug discovery today · 2014Review
- Examination of Huntington's disease in a Chinese family.Neural regeneration research · 2014Article
- Article
- Novel siRNA delivery strategy: a new "strand" in CNS translational medicine?Cellular and molecular life sciences : CMLS · 2014Review
- Epigenetic mechanisms of neurodegeneration in Huntington's disease.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2013Review
- Mechanisms of RNA-induced toxicity in CAG repeat disorders.Cell death & disease · 2013Review
- Repair of Mybpc3 mRNA by 5'-trans-splicing in a Mouse Model of Hypertrophic Cardiomyopathy.Molecular therapy. Nucleic acids · 2013Article
- Optimization of Ultrasound-mediated Anti-angiogenic Cancer Gene Therapy.Molecular therapy. Nucleic acids · 2013Article
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 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Huntington's disease (HD) is caused by an expansion of CAG triplets at the 5' end of the HD gene, which encodes a pathologically elongated polyglutamine stretch near the N-terminus of huntingtin. HD is an incurable autosomal-dominant neurodegenerative disease characterized by movement disorder, as well as emotional distress and dementia. The newly discovered roles of the non-coding small RNAs in specific degradation or translational suppression of the targeted mRNAs suggest a potential therapeutic approach of post-transcriptional gene silencing that targets the underlying disease etiology rather than the downstream pathological consequences. From pre-clinical trials in different HD animal models to cells from HD patients, small RNA interference has been applied to 'allele-non-specifically or allele-specifically' silence the mutant HD transgene or endogenous mutant HD allele. Silencing the mutant HD transgene significantly inhibits neurodegeneration, improves motor control, and extends survival of HD mice. With future improvement of mutant allele selectivity (preserving the expression of the neuroprotective wild-type allele), target specificity, efficacy and safety, as well as optimization of delivery methods, small non-coding RNA-based therapeutic applications will be a promising approach to treat HD.
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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.