Evidence map›Paper›PMID 29653083›Full record

ReviewBrain research2018

Aberrant RNA translation in fragile X syndrome: From FMRP mechanisms to emerging therapeutic strategies.

Anwesha Banerjee, Marius F Ifrim, Arielle N Valdez, Nisha Raj, Gary J Bassell

3 registry-linked trialsOpen access · hybridAbstract readReview
In one paragraph

Review in Brain research, 2018. 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 72 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
72citing papers in PubMed, 1 pooled it
11.6field-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

72 citing papers in PubMed, 1 synthesis or guideline pooled it, 115 citations in OpenAlex.

  1. Pooled it
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  3. International journal of molecular sciences · 2026
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12 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

5 authors at 1 institution in 1 country.

Anwesha BanerjeeDepartment of Cell Biology, Emory University School of Medicine, Atlanta, GA 30322, USA. Electronic address: anwesha.banerjee@emory.edu.
Marius F IfrimDepartment of Cell Biology, Emory University School of Medicine, Atlanta, GA 30322, USA.
Arielle N ValdezDepartment of Cell Biology, Emory University School of Medicine, Atlanta, GA 30322, USA.
Nisha RajDepartment of Cell Biology, Emory University School of Medicine, Atlanta, GA 30322, USA.
Gary J BassellDepartment of Cell Biology, Emory University School of Medicine, Atlanta, GA 30322, USA; Department of Neurology, Emory University School of Medicine, Atlanta, GA 30322, USA. Electronic address: gary.bassell@emory.edu.
Emory University · US

Funding

MEDICAL SCIENTIST TRAINING PROGRAMT32GM008169 · NIGMS · EMORY UNIVERSITY · PI GROSS, ROBERT E · 1987 to 2021
$20.3M
FMRP Mechanism and FunctionR01MH109026 · NIMH · EMORY UNIVERSITY · PI BASSELL, GARY J · 2016 to 2020
$2.0M
Mechanisms of FMRP mediated translational repressionF31NS101932 · NINDS · EMORY UNIVERSITY · PI VALDEZ, ARIELLE · 2017 to 2019
$134k
NIGMS NIH HHS T32 GM008169NIMH NIH HHS R01 MH109026NINDS NIH HHS F31 NS101932
6 · The paper itself

Abstract

Research in the past decades has unfolded the multifaceted role of Fragile X mental retardation protein (FMRP) and how its absence contributes to the pathophysiology of Fragile X syndrome (FXS). Excess signaling through group 1 metabotropic glutamate receptors is commonly observed in mouse models of FXS, which in part is attributed to dysregulated translation and downstream signaling. Considering the wide spectrum of cellular and physiologic functions that loss of FMRP can affect in general, it may be advantageous to pursue disease mechanism based treatments that directly target translational components or signaling factors that regulate protein synthesis. Various FMRP targets upstream and downstream of the translational machinery are therefore being investigated to further our understanding of the molecular mechanism of RNA and protein synthesis dysregulation in FXS as well as test their potential role as therapeutic interventions to alleviate FXS associated symptoms. In this review, we will broadly discuss recent advancements made towards understanding the role of FMRP in translation regulation, new pre-clinical animal models with FMRP targets located at different levels of the translational and signal transduction pathways for therapeutic intervention as well as future use of stem cells to model FXS associated phenotypes.

Indexed as

AnimalsDendritesDisease Models, AnimalFragile X Messenger Ribonucleoprotein 1Fragile X SyndromeGene Expression RegulationHumansProtein BiosynthesisReceptors, Metabotropic GlutamateRNA, MessengerSignal TransductionFragile X Messenger Ribonucleoprotein 1metabotropic glutamate receptor type 1Receptors, Metabotropic GlutamateRNA, MessengerDendritic spineFragile X syndromeiPSCmRNA localizationmRNA translationRNA binding protein

Identifiers

PMID29653083
PMCPMC7377270
OpenAlexW2797244528

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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 Socratic graph.