ReviewHuman molecular genetics2010
Huntington's disease: progress toward effective disease-modifying treatments and a cure.
Review in Human molecular genetics, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 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
33 citing papers in PubMed.
- DurableMolecular therapy. Nucleic acids · 2025Article
- Unraveling the Mechanisms of Cannabidiol's Pharmacological Actions: A Comprehensive Research Overview.Topics in current chemistry (Cham) · 2024Review
- The Therapeutic Potential and Molecular Mechanisms Underlying the Neuroprotective Effects of SativexMini reviews in medicinal chemistry · 2024Review
- Allele-specific silencing of the gain-of-function mutation in Huntington's disease using CRISPR/Cas9.JCI insight · 2022Article
- PAM-altering SNP-based allele-specific CRISPR-Cas9 therapeutic strategies for Huntington's disease.Molecular therapy. Methods & clinical development · 2022Article
- Rescue of BDNF expression by the thalamic parafascicular nucleus with chronic treatment with the mGluR2/3 agonist LY379268 may contribute to the LY379268 rescue of enkephalinergic striatal projection neurons in R6/2 Huntington's disease mice.Neuroscience letters · 2021Article
- Targeting Mitochondrial Network Disorganization is Protective inAging and disease · 2021Article
- Precise CAG repeat contraction in a Huntington's Disease mouse model is enabled by gene editing with SpCas9-NG.Communications biology · 2021Article
- Common Factors in Neurodegeneration: A Meta-Study Revealing Shared Patterns on a Multi-Omics Scale.Cells · 2020Article
- Different Clinical Contexts of Use of Blood Neurofilament Light Chain Protein in the Spectrum of Neurodegenerative Diseases.Molecular neurobiology · 2020Review
- Recent advances in molecular therapies for neurological disease: triplet repeat disorders.Human molecular genetics · 2019Review
- Evaluating the current state of the art of Huntington disease research: a scientometric analysis.Brazilian journal of medical and biological research = Revista brasileira de pesquisas medicas e biologica · 2018Review
- Striatal Mutant Huntingtin Protein Levels Decline with Age in Homozygous Huntington's Disease Knock-In Mouse Models.Journal of Huntington's disease · 2018Article
- Effects of a Sativex-Like Combination of Phytocannabinoids on Disease Progression in R6/2 Mice, an Experimental Model of Huntington's Disease.International journal of molecular sciences · 2017Article
- CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Allele-specific regulation of mutant Huntingtin by Wig1, a downstream target of p53.Human molecular genetics · 2016Article
- Genetically engineered livestock for biomedical models.Transgenic research · 2016Review
- Huntington Disease in Asia.Chinese medical journal · 2015Review
- [Huntington's disease].Der Nervenarzt · 2015Review
- Neuroprotective properties of cannabigerol in Huntington's disease: studies in R6/2 mice and 3-nitropropionate-lesioned mice.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2015Article
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Huntington's disease (HD) is caused by a dominant mutation in HTT, the HD gene. This discovery opens possibilities for treatment based on silencing of the disease-causing allele or with compounds that reduce the production of disease-causing mRNA and/or protein. Although additional developments are needed related to the delivery of gene silencing and discovery and development of drugs that reduce disease-causing gene products, these treatments are predicted to be effective since they act by reducing the source of toxicity. The identification of therapies that act by blocking toxicity is conceptually more complicated, as this requires an accurate understanding of the cellular location and the specific molecular dysfunctions that cause the phenotypes of HD, which is not yet available. Though challenges remain, significant progress has been made. Effective disease-modifying treatments will soon be tested and may lead to disease-altering therapies.
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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.