Evidence map›Paper›PMID 23584741›Full record

ReviewNature neuroscience2013

The translation of translational control by FMRP: therapeutic targets for FXS.

Jennifer C Darnell, Eric Klann

3 registry-linked trialsAbstract readReview
In one paragraph

Review in Nature neuroscience, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 285 papers.

0numbers the graph read from it
0cells of the map it votes in
285citing papers in PubMed
33.2field-weighted citation impact, top 1% of its field
1 · What the graph read from it

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.

2 · The registry

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.

NCT06480968 phase1completednot on this mapstarted 2023, after this paper: background citation

Safety and Tolerability of CTH120, First-in-human Phase I Study Encompassing Three Parts: Single and Multiple Ascending Doses and Potential Food Interaction

TypeinterventionalSponsorConnecta Therapeutics, S.L.Ran2023 to 2024Enrolled76ConditionsFragile X SyndromeArmsCTH120, Placebo
NCT06957054 completednot on this mapstarted 2024, after this paper: background citation

Assessment of Neural Biomarkers in Adult Subjects With Fragile X Syndrome and Typically Developing Subjects

TypeobservationalSponsorConnecta Therapeutics, S.L.Ran2024 to 2025Enrolled20ConditionsFragile X Syndrome (FXS), Neurotypical AdultsArmsAny
NCT07654114 phase2recruitingnot on this mapstarted 2026, after this paper: background citation

Evaluation of the Safety, Tolerability, and Effectiveness of CTH120 in Adult Males With Fragile X Syndrome

TypeinterventionalSponsorConnecta Therapeutics, S.L.Ran2026 to 2027Enrolled30ConditionsFragile X Syndrome (FXS), Fragile X SyndromeArmsCTH120, CTH120 placebo hard capsules
3 · Its place in the literature

Who cites it

285 citing papers in PubMed, 492 citations in OpenAlex.

  1. Trial
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  4. mSphere · 2026
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  8. The mMolecular psychiatry · 2026
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225 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors at 2 institutions in 1 country.

Jennifer C DarnellLaboratory of Molecular Neuro-Oncology, The Rockefeller University, New York, New York, USA.
Eric Klann
New York University · USRockefeller University · US

Funding

Translation Regulation in Hippocampal LTP and LTDR01NS047384 · NINDS · NEW YORK UNIVERSITY · PI KLANN, ERIC · 2005 to 2020
$5.2M
IDENTIFICATION OF FMRP TARGET RNASR01HD040647 · NICHD · ROCKEFELLER UNIVERSITY · PI DARNELL, JENNIFER C · 2001 to 2017
$4.5M
NICHD NIH HHS HD040647NICHD NIH HHS R01 HD040647NINDS NIH HHS NS047384NINDS NIH HHS R01 NS047384
6 · The paper itself

Abstract

De novo protein synthesis is necessary for long-lasting modifications in synaptic strength and dendritic spine dynamics that underlie cognition. Fragile X syndrome (FXS), characterized by intellectual disability and autistic behaviors, holds promise for revealing the molecular basis for these long-term changes in neuronal function. Loss of function of the fragile X mental retardation protein (FMRP) results in defects in synaptic plasticity and cognition in many models of the disease. FMRP is a polyribosome-associated RNA-binding protein that regulates the synthesis of a set of plasticity-reated proteins by stalling ribosomal translocation on target mRNAs. The recent identification of mRNA targets of FMRP and its upstream regulators, and the use of small molecules to stall ribosomes in the absence of FMRP, have the potential to be translated into new therapeutic avenues for the treatment of FXS.

Indexed as

Fragile X SyndromeAnimalsFragile X Messenger Ribonucleoprotein 1HumansIntellectual DisabilityNeuronal PlasticityProtein BiosynthesisReceptors, Metabotropic GlutamateRNA, MessengerFragile X Messenger Ribonucleoprotein 1Receptors, Metabotropic GlutamateRNA, Messenger

Identifiers

PMID23584741
PMCPMC3999698
OpenAlexW2001215117

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

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.