Contact and support
Need help, have a question, or want to contact the ResearchHub team?
© 2026 ResearchHub. Built for responsible scholarly connection.
Privacy-aware community infrastructureResearch graph
References from Therapeutic approaches for the future treatment of Fragile X. Local targets link to admitted publications; unresolved targets remain external evidence.
Fragile X syndrome: a review of associated medical problems
10.1542/peds.2013-4301 · 2014 · External reference
Identification of a gene (FMR-1) containing a CGG repeat coincident with a breakpoint cluster region exhibiting length variation in fragile X syndrome
10.1016/0092-8674(91)90397-h · 1991 · External reference
New perspectives on the biology of fragile X syndrome
10.1016/j.gde.2012.02.002 · 2012 · External reference
The pathophysiology of fragile X syndrome
10.1146/annurev.genom.8.080706.092249 · 2007 · External reference
FMR1 and the fragile X syndrome: human genome epidemiology review
10.1097/00125817-200109000-00006 · 2001 · External reference
Prevalence of carriers of premutation-size alleles of the FMRI gene — and implications for the population genetics of the fragile X syndrome
1995 · External reference
Epidemiology of fragile X syndrome: a systematic review and meta-analysis
10.1002/ajmg.a.36511 · 2014 · External reference
Translating molecular advances in fragile X syndrome into therapy: a review
10.4088/jcp.13r08714 · 2014 · External reference
Fragile X mental retardation protein: past, present and future
10.2174/138920312801619420 · 2012 · External reference
A point mutation in the FMR-1 gene associated with fragile X mental retardation
10.1038/ng0193-31 · 1993 · External reference
Novel point mutation within intron 10 of FMR-1 gene causing fragile X syndrome
10.1002/(sici)1098-1004(1997)10:5<393::aid-humu10>3.0.co;2-v · 1997 · External reference
Fragile X mental retardation syndrome: structure of the KH1-KH2 domains of fragile X mental retardation protein
10.1016/j.str.2007.06.022 · 2007 · External reference
Characterization of FMR1 proteins isolated from different tissues
10.1093/hmg/4.5.895 · 1995 · External reference
FMRP associates with polyribosomes as an mRNP, and the I304N mutation of severe fragile X syndrome abolishes this association
10.1016/s1097-2765(00)80012-x · 1997 · External reference
Kissing complex RNAs mediate interaction between the Fragile-X mental retardation protein KH2 domain and brain polyribosomes
10.1101/gad.1276805 · 2005 · External reference
Identification of novel FMR1 variants by massively parallel sequencing in developmentally delayed males
10.1002/ajmg.a.33626 · 2010 · External reference
Evidence that methylation of the FMR-I locus is responsible for variable phenotypic expression of the fragile X syndrome
1993 · External reference
Phenotypic variation and FMRP levels in fragile X
10.1002/mrdd.20006 · 2004 · External reference
Fragile X syndrome and X-linked intellectual disability
2013 · External reference
The fragile X syndrome-autism comorbidity: what do we really know?
10.3389/fgene.2014.00355 · 2014 · External reference
Autism and fragile X syndrome
10.1055/s-0034-1386764 · 2014 · External reference
Origins of epilepsy in fragile X syndrome
10.1111/j.1535-7511.2009.01309.x · 2009 · External reference
The FMR-1 protein is cytoplasmic, most abundant in neurons and appears normal in carriers of a fragile X premutation
10.1038/ng0893-335 · 1993 · External reference
Structural studies of the tandem Tudor domains of fragile X mental retardation related proteins FXR1 and FXR2
10.1371/journal.pone.0013559 · 2010 · External reference
Structure-function studies of FMRP RGG peptide recognition of an RNA duplex–quadruplex junction
10.1038/nsmb.2064 · 2011 · External reference
The structure of the N-terminal domain of the fragile X mental retardation protein: a platform for protein–protein interaction
10.1016/j.str.2005.09.018 · 2006 · External reference
The unstable repeats — three evolving faces of neurological disease
10.1016/j.neuron.2013.02.022 · 2013 · External reference
Fragile X syndrome: from protein function to therapy
10.1002/ajmg.a.36241 · 2013 · External reference
Structure and function of KH domains
10.1111/j.1742-4658.2008.06411.x · 2008 · External reference
The fragile X syndrome protein represses activity-dependent translation through CYFIP1, a new 4E-BP
10.1016/j.cell.2008.07.031 · 2008 · External reference
Fragile X syndrome: mechanistic insights and therapeutic avenues regarding the role of potassium channels
10.1016/j.conb.2012.03.010 · 2012 · External reference
FMRP stalls ribosomal translocation on mRNAs linked to synaptic function and autism
10.1016/j.cell.2011.06.013 · 2011 · External reference
Fragile X mental retardation protein interactions with the microtubule associated protein 1B RNA
10.1261/rna.1100708 · 2008 · External reference
Evidence for a fragile X mental retardation protein-mediated translational switch in metabotropic glutamate receptor-triggered Arc translation and long-term depression
10.1523/jneurosci.4650-11.2012 · 2012 · External reference
Taking STEPs forward to understand fragile X syndrome
10.1007/978-3-642-21649-7_12 · 2012 · External reference
Genetic manipulation of STEP reverses behavioral abnormalities in a fragile X syndrome mouse model
10.1111/j.1601-183x.2012.00781.x · 2012 · External reference
Fragile X mental retardation protein is required for synapse elimination by the activity-dependent transcription factor MEF2
10.1016/j.neuron.2010.03.017 · 2010 · External reference
Roles of fragile X mental retardation protein in dopaminergic stimulation-induced synapse-associated protein synthesis and subsequent alpha-amino-3-hydroxyl-5-methyl-4-isoxazole-4-propionate (AMPA) receptor internalization
10.1074/jbc.m110.116293 · 2010 · External reference
FMRP regulates neurotransmitter release and synaptic information transmission by modulating action potential duration via BK channels
10.1016/j.neuron.2012.12.018 · 2013 · External reference
Advances in the treatment of fragile X syndrome
10.1542/peds.2008-0317 · 2009 · External reference
Behavioral intervention for problem behavior in children with fragile X syndrome
10.1352/1944-7558-116.6.457 · 2011 · External reference
Psychopharmacology in fragile X syndrome — present and future
10.1002/mrdd.20007 · 2004 · External reference
Health supervision for children with fragile X syndrome
10.1542/peds.2010-3500 · 2011 · External reference
Medication utilization for targeted symptoms in children and adults with fragile X syndrome: US survey
10.1097/dbp.0b013e318236c0e1 · 2012 · External reference
Neuropsychiatric symptoms of fragile X syndrome: pathophysiology and pharmacotherapy
10.2165/00023210-200418110-00001 · 2004 · External reference
Sertraline may improve language developmental trajectory in young children with fragile X syndrome: a retrospective chart review
2012 · External reference
Risperidone in children with autism: randomized, placebo-controlled, double-blind study
10.1177/08830738060210060801 · 2006 · External reference
Seizures in fragile X syndrome: characteristics and comorbid diagnoses
10.1352/1944-7558-115.6.461 · 2010 · External reference
New insights into the molecular pathophysiology of fragile X syndrome and therapeutic perspectives from the animal model
10.1016/j.biocel.2014.05.004 · 2014 · External reference
Dysregulation of group-I metabotropic glutamate (mGlu) receptor mediated signalling in disorders associated with intellectual disability and autism
10.1016/j.neubiorev.2014.02.003 · 2014 · External reference
The mGluR theory of fragile X mental retardation
10.1016/j.tins.2004.04.009 · 2004 · External reference
GABA system dysfunction in autism and related disorders: from synapse to symptoms
10.1016/j.neubiorev.2012.07.005 · 2012 · External reference
Involvement and therapeutic potential of the GABAergic system in the fragile X syndrome
10.1100/tsw.2010.211 · 2010 · External reference
GABAergic signaling as therapeutic target for autism spectrum disorders
10.3389/fped.2014.00070 · 2014 · External reference
GABAergic and glycinergic interneuron expression during spinal cord development: dynamic interplay between inhibition and excitation in the control of ventral network outputs
10.1016/j.pneurobio.2009.06.001 · 2009 · External reference
Pharmacological treatment of fragile X syndrome with GABAergic drugs in a knockout mouse model
10.1016/j.bbr.2012.01.031 · 2012 · External reference
Insights into GABAergic system deficits in fragile X syndrome lead to clinical trials
10.1016/j.neuropharm.2014.06.028 · 2015 · External reference
Targeted treatments in autism and fragile X syndrome
10.1016/j.rasd.2012.05.007 · 2012 · External reference
A pilot open label, single dose trial of fenobam in adults with fragile X syndrome
10.1136/jmg.2008.063701 · 2009 · External reference
Epigenetic modification of the FMR1 gene in fragile X syndrome is associated with differential response to the mGluR5 antagonist AFQ056
10.1126/scitranslmed.3001708 · 2011 · External reference
Fragile X syndrome: from targets to treatments
10.1016/j.neuropharm.2012.11.028 · 2013 · External reference
STX209 (arbaclofen) for autism spectrum disorders: an 8-week open-label study
10.1007/s10803-013-1963-z · 2014 · External reference
Clinical potential, safety, and tolerability of arbaclofen in the treatment of autism spectrum disorder
10.2147/dhps.s39595 · 2014 · External reference
Fragile X syndrome and targeted treatment trials
10.1007/978-3-642-21649-7_17 · 2012 · External reference
Glycogen synthase kinase-3 as a therapeutic target for cognitive dysfunction in neuropsychiatric disorders
2014 · External reference
Glycogen synthase kinase-3: a promising therapeutic target for fragile X syndrome
10.3389/fnmol.2011.00035 · 2011 · External reference
Glycogen synthase kinase-3 inhibitors: rescuers of cognitive impairments
10.1016/j.pharmthera.2013.07.010 · 2014 · External reference
The role of glycogen synthase kinase-3 signaling in neurodevelopment and fragile X syndrome
2012 · External reference
Elevated glycogen synthase kinase-3 activity in fragile X mice: key metabolic regulator with evidence for treatment potential
10.1016/j.neuropharm.2008.09.017 · 2009 · External reference
Evidence of reactive astrocytes but not peripheral immune system activation in a mouse model of Fragile X syndrome
10.1016/j.bbadis.2010.06.015 · 2010 · External reference
Fragile X mental retardation protein regulates proliferation and differentiation of adult neural stem/progenitor cells
10.1371/journal.pgen.1000898 · 2010 · External reference
Lithium ameliorates altered glycogen synthase kinase-3 and behavior in a mouse model of fragile X syndrome
10.1016/j.bcp.2009.09.023 · 2010 · External reference
Lithium ameliorates open-field and elevated plus maze behaviors, and brain phospho-glycogen synthase kinase 3-beta expression in fragile X syndrome model mice
2013 · External reference
Molecular actions and therapeutic potential of lithium in preclinical and clinical studies of CNS disorders
10.1016/j.pharmthera.2010.07.006 · 2010 · External reference
Treatment with valproic acid ameliorates ADHD symptoms in fragile X syndrome boys
10.1002/ajmg.a.33484 · 2010 · External reference
Lithium: a review of pharmacology, clinical uses, and toxicity
10.1016/j.ejphar.2014.06.042 · 2014 · External reference
Inhibition of GSK3beta improves hippocampus-dependent learning and rescues neurogenesis in a mouse model of fragile X syndrome
10.1093/hmg/ddr501 · 2012 · External reference
Glycogen synthase kinase-3 inhibitors reverse deficits in long-term potentiation and cognition in fragile X mice
10.1016/j.biopsych.2013.08.003 · 2014 · External reference
Neuron-specific regulation of class I PI3K catalytic subunits and their dysfunction in brain disorders
10.3389/fnmol.2014.00012 · 2014 · External reference
Excess phosphoinositide 3-kinase subunit synthesis and activity as a novel therapeutic target in fragile X syndrome
10.1523/jneurosci.0402-10.2010 · 2010 · External reference
Identification of fragile x syndrome specific molecular markers in human fibroblasts: a useful model to test the efficacy of therapeutic drugs
10.1002/humu.22699 · 2014 · External reference
Excess protein synthesis in FXS patient lymphoblastoid cells can be rescued with a p110beta-selective inhibitor
10.2119/molmed.2011.00363 · 2012 · External reference
Novel approaches to inhibitor design for the p110beta phosphoinositide 3-kinase
10.1016/j.tips.2012.12.004 · 2013 · External reference
Deregulated mTOR-mediated translation in intellectual disability
10.1016/j.pneurobio.2012.01.005 · 2012 · External reference
Altered mTOR signaling and enhanced CYFIP2 expression levels in subjects with fragile X syndrome
10.1111/j.1601-183x.2012.00768.x · 2012 · External reference
Dysregulation of mTOR signaling in fragile X syndrome
10.1523/jneurosci.3696-09.2010 · 2010 · External reference
The Akt-mTOR pathway in Down's syndrome: the potential use of rapamycin/rapalogs for treating cognitive deficits
10.2174/18715273113126660184 · 2014 · External reference
Targeting the endocannabinoid system in the treatment of fragile X syndrome
10.1038/nm.3127 · 2013 · External reference
Rapamycin improves sociability in the BTBR T(+)Itpr3(tf)/J mouse model of autism spectrum disorders
10.1016/j.brainresbull.2013.11.005 · 2014 · External reference
mTOR inhibition: from aging to autism and beyond
2013 · External reference
Mitogen-activated protein kinases in synaptic plasticity and memory
10.1016/j.conb.2004.04.001 · 2004 · External reference
Zinc and the ERK kinases in the developing brain
10.1007/s12640-011-9291-6 · 2012 · External reference
Activation of the extracellular signal-regulated kinase pathway contributes to the behavioral deficit of fragile X-syndrome
10.1111/j.1471-4159.2012.07722.x · 2012 · External reference
Physiological activation of synaptic Rac>PAK (p-21 activated kinase) signaling is defective in a mouse model of fragile X syndrome
10.1523/jneurosci.1077-10.2010 · 2010 · External reference
Inhibitors of p21-activated kinases (PAKs)
2014 · External reference
PAKs inhibitors ameliorate schizophrenia-associated dendritic spine deterioration in vitro and in vivo during late adolescence
10.1073/pnas.1321109111 · 2014 · External reference
PAK in Alzheimer disease, Huntington disease and X-linked mental retardation
10.4161/cl.21602 · 2012 · External reference
Inhibition of p21-activated kinase rescues symptoms of fragile X syndrome in mice
10.1073/pnas.0705003104 · 2007 · External reference
Rescue of fragile X syndrome phenotypes in Fmr1 KO mice by the small-molecule PAK inhibitor FRAX486
10.1073/pnas.1219383110 · 2013 · External reference
Multiple roles for frequenin/NCS-1 in synaptic function and development
10.1007/s12035-012-8250-4 · 2012 · External reference
Neuronal calcium sensor-1 regulation of calcium channels, secretion, and neuronal outgrowth
10.1007/s10571-010-9588-7 · 2010 · External reference
Frequenin/NCS-1 and the Ca2+-channel alpha1-subunit co-regulate synaptic transmission and nerve-terminal growth
10.1242/jcs.055095 · 2009 · External reference
A postsynaptic signaling pathway that may account for the cognitive defect due to IL1RAPL1 mutation
10.1016/j.cub.2009.12.030 · 2010 · External reference
Mutations in the calcium-related gene IL1RAPL1 are associated with autism
10.1093/hmg/ddn300 · 2008 · External reference
The guanine-exchange factor Ric8a binds the calcium sensor NCS-1 to regulate synapse number and probability of release
10.1242/jcs.152603 · 2014 · External reference
Bidirectional regulation of dendritic voltage-gated potassium channels by the fragile X mental retardation protein
10.1016/j.neuron.2011.09.033 · 2011 · External reference
Fragile X mental retardation protein is required for rapid experience-dependent regulation of the potassium channel Kv3.1b
10.1523/jneurosci.1125-10.2010 · 2010 · External reference
Potassium channel modulation and auditory processing
10.1016/j.heares.2011.03.004 · 2010 · External reference
Fragile X mental retardation protein controls gating of the sodium-activated potassium channel Slack
10.1038/nn.2563 · 2011 · External reference
Fragile X mental retardation protein targets G quartet mRNAs important for neuronal function
10.1016/s0092-8674(01)00566-9 · 2001 · External reference
Regulation of the timing of MNTB neurons by short-term and long-term modulation of potassium channels
10.1016/j.heares.2004.11.023 · 2005 · External reference
Fragile X mental retardation protein regulates protein expression and mRNA translation of the potassium channel Kv4.2
10.1523/jneurosci.6661-10.2011 · 2011 · External reference
Hypersensitivity to mGluR5 and ERK1/2 leads to excessive protein synthesis in the hippocampus of a mouse model of fragile X syndrome
10.1523/jneurosci.3888-10.2010 · 2010 · External reference
Lovastatin inhibits the extracellular-signal-regulated kinase pathway in immortalized rat brain neuroblasts
10.1042/bj20060731 · 2007 · External reference
Fragile X syndrome therapeutics: translation, meet translational medicine
10.1016/j.neuron.2013.01.009 · 2013 · External reference
Lovastatin corrects excess protein synthesis and prevents epileptogenesis in a mouse model of fragile X syndrome
10.1016/j.neuron.2012.01.034 · 2013 · External reference
Effect of lovastatin on behavior in children and adults with fragile X syndrome: an open-label study
10.1002/ajmg.a.36750 · 2014 · External reference
Cholesterol levels in Fragile X syndrome
2014 · External reference
Lipid rafts: a signaling platform linking cholesterol metabolism to synaptic deficits in autism spectrum disorders
10.3389/fnbeh.2014.00104 · 2014 · External reference
Extracellular proteolysis by matrix metalloproteinase-9 drives dendritic spine enlargement and long-term potentiation coordinately
10.1073/pnas.0807248105 · 2008 · External reference
Influence of matrix metalloproteinase MMP-9 on dendritic spine morphology
10.1242/jcs.090852 · 2011 · External reference
Minocycline promotes dendritic spine maturation and improves behavioural performance in the fragile X mouse model
10.1136/jmg.2008.061796 · 2009 · External reference
Important role of matrix metalloproteinase 9 in epileptogenesis
10.1083/jcb.200708213 · 2008 · External reference
Tassone F: High MMP-9 activity levels in fragile X syndrome are lowered by minocycline
10.1002/ajmg.a.36023 · 2013 · External reference
Matrix metalloproteinases and minocycline: therapeutic avenues for fragile X syndrome
10.1155/2012/124548 · 2012 · External reference
Open-label add-on treatment trial of minocycline in fragile X syndrome
10.1186/1471-2377-10-91 · 2010 · External reference
Side effects of minocycline treatment in patients with fragile X syndrome and exploration of outcome measures
10.1352/1944-7558-115.5.433 · 2010 · External reference
Involvement of cholinergic and GABAergic systems in the fragile X knockout mice
10.1016/s0306-4522(03)00103-9 · 2003 · External reference
Modulation of behavioral phenotypes by a muscarinic M1 antagonist in a mouse model of fragile X syndrome
10.1007/s00213-011-2276-6 · 2011 · External reference
The modulation of fragile X behaviors by the muscarinic M4 antagonist, tropicamide
10.1037/a0025202 · 2011 · External reference
Genetic reduction of muscarinic M4 receptor modulates analgesic response and acoustic startle response in a mouse model of fragile X syndrome (FXS)
10.1016/j.bbr.2011.11.018 · 2012 · External reference
Multiple Gq-coupled receptors converge on a common protein synthesis-dependent long-term depression that is affected in fragile X syndrome mental retardation
10.1523/jneurosci.2266-07.2007 · 2007 · External reference
Enhanced markers of oxidative stress, altered antioxidants and NADPH-oxidase activation in brains from Fragile X mental retardation 1-deficient mice, a pathological model for Fragile X syndrome
10.1111/j.1460-9568.2007.05939.x · 2007 · External reference
A novel function for fragile X mental retardation protein in translational activation
10.1371/journal.pbio.1000016 · 2009 · External reference
Alpha-tocopherol protects against oxidative stress in the fragile X knockout mouse: an experimental therapeutic approach for the Fmr1 deficiency
10.1038/npp.2008.152 · 2009 · External reference
Protective effects of melatonin against oxidative stress in Fmr1 knockout mice: a therapeutic research model for the fragile X syndrome
10.1111/j.1600-079x.2008.00653.x · 2009 · External reference
Targeting the endocannabinoid system in the treatment of fragile X syndrome
10.1038/nm.3127 · 2013 · External reference
Abnormal mGlu 5 receptor/endocannabinoid coupling in mice lacking FMRP and BC1 RNA
10.1038/npp.2010.19 · 2010 · External reference
Fmr1 deletion enhances and ultimately desensitizes CB(1) signaling in autaptic hippocampal neurons
10.1016/j.nbd.2013.04.002 · 2013 · External reference
Enhanced endocannabinoid signaling elevates neuronal excitability in fragile X syndrome
10.1523/jneurosci.0795-10.2010 · 2010 · External reference
Uncoupling of the endocannabinoid signalling complex in a mouse model of fragile X syndrome
10.1038/ncomms2045 · 2012 · External reference
Selective blockade of 2-arachidonoylglycerol hydrolysis produces cannabinoid behavioral effects
10.1038/nchembio.129 · 2009 · External reference
Peroxisome proliferator activated receptor gamma (PPARgamma) as a therapeutic target for improvement of cognitive performance in Fragile-X
10.1016/j.mehy.2013.12.012 · 2014 · External reference
