ArticleScientific reports2022
Fragment-based virtual screening identifies a first-in-class preclinical drug candidate for Huntington's disease.
Article in Scientific reports, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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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.
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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.
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Who cites it
7 citing papers in PubMed.
- Prediction of SafD adhesin strong binding peptides for pilus proteins assembly suppression in the prevention ofIn silico pharmacology · 2025Article
- Review
- Correlation of protein binding pocket properties with hits' chemistries used in generation of ultra-large virtual libraries.Journal of computer-aided molecular design · 2024Article
- Fragment-based drug discovery for disorders of the central nervous system: designing better drugs piece by piece.Frontiers in chemistry · 2024Review
- HD_BPMDS: a curated binary pattern multitarget dataset of Huntington's disease-targeting agents.Journal of cheminformatics · 2023Article
- Huntington's Disease Drug Development: A Phase 3 Pipeline Analysis.Pharmaceuticals (Basel, Switzerland) · 2023Review
- Emerging Therapies for Huntington's Disease - Focus on N-Terminal Huntingtin and Huntingtin Exon 1.Biologics : targets & therapy · 2022Review
Corrections and comments
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Authors and funding
6 authors.
Funding
Abstract
Currently, there are no therapies available to modify the disease progression of Huntington's disease (HD). Recent clinical trial failures of antisense oligonucleotide candidates in HD have demonstrated the need for new therapeutic approaches. Here, we developed a novel in-silico fragment scanning approach across the surface of mutant huntingtin (mHTT) polyQ and predicted four hit compounds. Two rounds of compound analoging using a strategy of testing structurally similar compounds in an affinity assay rapidly identified GLYN122. In vitro, GLYN122 directly binds and reduces mHTT and induces autophagy in neurons. In vivo, our results confirm that GLYN122 can reduce mHTT in the cortex and striatum of the R/2 mouse model of Huntington's disease and subsequently improve motor symptoms. Thus, the in-vivo pharmacology profile of GLYN122 is a potential new preclinical candidate for the treatment of HD.
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Registered trials
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