ReviewNature genetics2025
Therapeutic strategies for fragile X syndrome and implications for other gene-silencing disorders.
Review in Nature genetics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 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
4 citing papers in PubMed.
- RNA-Based Therapeutics in Genetic Neurodevelopmental Disorders: Bridging Molecular Genetics and Precision Medicine.International journal of molecular sciences · 2026Review
- FYN, a Novel Target of Fragile X Mental Retardation Protein, Potentially Underlies ERK1/2 Hyperactivation in Fragile X Syndrome.Molecular neurobiology · 2026Article
- Cerebellar microglia-derived IL-17A mitigates autism-related behavioral and synaptic deficits.Molecular psychiatry · 2026Article
- Artificial intelligence revolutionizing CNS drug discovery and development.Drug discovery today · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
Gene-silencing disorders, of which fragile X syndrome (FXS) is the most prevalent, are diseases caused by a blockade of gene transcription, usually due to DNA hypermethylation. FXS is a common form of inherited intellectual disability and autism. Unlike most hereditary diseases driven by mutations within the protein-coding region of a gene, FXS is caused by a trinucleotide expansion in the 5'-untranslated region of the FMR1 gene, leading to hypermethylation and transcriptional silencing. Modeling FXS with human pluripotent stem cells offers a clinically relevant platform to study disease mechanisms and explore potential therapies through reactivating FMR1 expression by genetic and epigenetic means or through drug screening. This Perspective reviews the various cellular models and therapeutic strategies proposed over the past decade, highlighting their potential to advance the treatment of FXS. We also discuss the benefits and challenges of gene activation therapies, drawing comparisons with other gene-silencing disorders, including imprinting diseases and X-linked disorders.
Indexed as
Identifiers
40676228What 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.