The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities

Role of phosphodiesterases in the pathophysiology of neurodevelopmental disorders

(2021) Sébastien Delhaye et al.   MOLECULAR PSYCHIATRY

Trends in peptide drug discovery

(2021) Markus Muttenthaler et al.   NATURE REVIEWS DRUG DISCOVERY

The molecular biology of FMRP: new insights into fragile X syndrome

(2021) Joel D. Richter et al.   NATURE REVIEWS NEUROSCIENCE

Inhibition of phosphodiesterase-4D in adults with fragile X syndrome: a randomized, placebo-controlled, phase 2 clinical trial

(2021) Elizabeth M. Berry-Kravis et al.   NATURE MEDICINE

The safety and efficacy of metformin in fragile X syndrome: An open-label study

(2021) Mélodie Proteau-Lemieux et al.   PROGRESS IN NEURO-PSYCHOPHARMACOLOGY & BIOLOGICAL PSYCHIATRY

Fragile X syndrome and associated disorders: Clinical aspects and pathology

(2020) Maria Jimena Salcedo-Arellano et al.   NEUROBIOLOGY OF DISEASE

Reactivation of FMR1 gene expression is a promising strategy for fragile X syndrome therapy

(2020) Ekaterina M. Shitik et al.   GENE THERAPY

Peptide Therapeutics 2.0

(2020) Beatriz G. de la Torre et al.   MOLECULES

Correlation between hemolytic activity, cytotoxicity and systemic in vivo toxicity of synthetic antimicrobial peptides

(2020) Ines Greco et al.   Scientific Reports

Bioinformatics and Biosimulations as Toolbox for Peptides and Peptidomimetics Design: Where Are We?

(2020) Ilda D’Annessa et al.   Frontiers in Molecular Biosciences

Inhibition of PDE2 and PDE4 synergistically improves memory consolidation processes

(2020) Dean Paes et al.   NEUROPHARMACOLOGY

Peptidomimetics: A Synthetic Tool for Inhibiting Protein–Protein Interactions in Cancer

(2019) Lloyd Mabonga et al.   International Journal of Peptide Research and Therapeutics

A Comprehensive Review on Current Advances in Peptide Drug Development and Design

(2019) Andy Chi-Lung Lee et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Design and Synthesis of Selective Phosphodiesterase 4D (PDE4D) Allosteric Inhibitors for the Treatment of Fragile X Syndrome and Other Brain Disorders

(2019) Mark E. Gurney et al.   JOURNAL OF MEDICINAL CHEMISTRY

Incomplete silencing of full mutation alleles in males with fragile X syndrome is associated with autistic features

(2019) Emma K. Baker et al.   Molecular Autism

Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin‐two cases

(2019) Dragana Protic et al.   Molecular Genetics & Genomic Medicine

A phase 1/2, open-label assessment of the safety, tolerability, and efficacy of transdermal cannabidiol (ZYN002) for the treatment of pediatric fragile X syndrome

(2019) Helen Heussler et al.   Journal of Neurodevelopmental Disorders

Metformin treatment in young children with fragile X syndrome

(2019) Hazel Maridith B. Biag et al.   Molecular Genetics & Genomic Medicine

The loss of β adrenergic receptor mediated release potentiation in a mouse model of fragile X syndrome

(2019) Nuria García-Font et al.   NEUROBIOLOGY OF DISEASE

Deciphering the mechanistic effects of eIF4E phosphorylation on mRNA‐cap recognition

(2019) Dilraj Lama et al.   PROTEIN SCIENCE

Peptides, Peptidomimetics, and Carbohydrate–Peptide Conjugates as Amyloidogenic Aggregation Inhibitors for Alzheimer’s Disease

(2018) Philip Ryan et al.   ACS Chemical Neuroscience

Peptide based therapeutics and their use for the treatment of neurodegenerative and other diseases

(2018) Mohammad Hassan Baig et al.   BIOMEDICINE & PHARMACOTHERAPY

OUP accepted manuscript

(2018)   CEREBRAL CORTEX

Structural motifs in eIF4G and 4E-BPs modulate their binding to eIF4E to regulate translation initiation in yeast

(2018) Stefan Grüner et al.   NUCLEIC ACIDS RESEARCH

HITS-CLIP in various brain areas reveals new targets and new modalities of RNA binding by fragile X mental retardation protein

(2018) Thomas Maurin et al.   NUCLEIC ACIDS RESEARCH

Handling FMRP and its molecular partners: Structural insights into Fragile X Syndrome

(2018) Ilda D'Annessa et al.   PROGRESS IN BIOPHYSICS & MOLECULAR BIOLOGY

Protein Synthesis Initiation in Eukaryotic Cells

(2018) William C. Merrick et al.   Cold Spring Harbor Perspectives in Biology

Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

(2018) Bumwhee Lee et al.   Nature Biomedical Engineering

Stress Odorant Sensory Response Dysfunction in Drosophila Fragile X Syndrome Mutants

(2018) Alaura Androschuk et al.   Frontiers in Molecular Neuroscience

Metformin for Treatment of Fragile X Syndrome and Other Neurological Disorders

(2018) Ilse Gantois et al.   Annual Review of Medicine

MiR-219 represses expression of dFMR1 in Drosophila melanogaster

(2018) Chao Wang et al.   LIFE SCIENCES

Intragenic FMR1 disease-causing variants: a significant mutational mechanism leading to Fragile-X syndrome

(2017) Angélique Quartier et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Short peptides interfering with signaling pathways as new therapeutic tools for cancer treatment

(2017) Aleksandra Ellert-Miklaszewska et al.   Future Medicinal Chemistry

Metformin ameliorates core deficits in a mouse model of fragile X syndrome

(2017) Ilse Gantois et al.   NATURE MEDICINE

Drug development for neurodevelopmental disorders: lessons learned from fragile X syndrome

(2017) Elizabeth M. Berry-Kravis et al.   NATURE REVIEWS DRUG DISCOVERY

MicroRNA therapeutics: towards a new era for the management of cancer and other diseases

(2017) Rajesha Rupaimoole et al.   NATURE REVIEWS DRUG DISCOVERY

Effect of the mGluR5-NAM Basimglurant on Behavior in Adolescents and Adults with Fragile X Syndrome in a Randomized, Double-Blind, Placebo-Controlled Trial: FragXis Phase 2 Results

(2017) Eriene A Youssef et al.   NEUROPSYCHOPHARMACOLOGY

Reducing eIF4E-eIF4G interactions restores the balance between protein synthesis and actin dynamics in fragile X syndrome model mice

(2017) Emanuela Santini et al.   Science Signaling

Rac1 GTPase activates the WAVE regulatory complex through two distinct binding sites

(2017) Baoyu Chen et al.   eLife

Reducing eIF4E-eIF4G interactions restores the balance between protein synthesis and actin dynamics in fragile X syndrome model mice

(2017) Emanuela Santini et al.   Science Signaling

Fragile X syndrome

(2017) Randi J. Hagerman et al.   Nature Reviews Disease Primers

Multiple Behavior Phenotypes of the Fragile-X Syndrome Mouse Model Respond to Chronic Inhibition of Phosphodiesterase-4D (PDE4D)

(2017) Mark E. Gurney et al.   Scientific Reports

Anti-Cancer Effect of Cap-Translation Inhibitor 4EGI-1 in Human Glioma U87 Cells: Involvement of Mitochondrial Dysfunction and ER Stress

(2016) Ming Wu et al.   CELLULAR PHYSIOLOGY AND BIOCHEMISTRY

Stapled peptide design: principles and roles of computation

(2016) Yaw Sing Tan et al.   DRUG DISCOVERY TODAY

Pharmacotherapy for Fragile X Syndrome: Progress to Date

(2016) Matthew H. Davenport et al.   DRUGS

A Randomized, Double-Blind, Placebo-Controlled Trial of Low-Dose Sertraline in Young Children With Fragile X Syndrome

(2016) Laura Greiss Hess et al.   JOURNAL OF DEVELOPMENTAL AND BEHAVIORAL PEDIATRICS

The Structures of eIF4E-eIF4G Complexes Reveal an Extended Interface to Regulate Translation Initiation

(2016) Stefan Grüner et al.   MOLECULAR CELL

Insulin signaling misregulation underlies circadian and cognitive deficits in a Drosophila fragile X model

(2016) R E Monyak et al.   MOLECULAR PSYCHIATRY

Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials

(2016) Elizabeth Berry-Kravis et al.   Science Translational Medicine

Transcriptional Reactivation of the FMR1 Gene. A Possible Approach to the Treatment of the Fragile X Syndrome

(2016) Elisabetta Tabolacci et al.   Genes

Reactivation of FMR1 by CRISPR/Cas9-Mediated Deletion of the Expanded CGG-Repeat of the Fragile X Chromosome

(2016) Nina Xie et al.   PLoS One

Intranasal Delivery of Proteins and Peptides in the Treatment of Neurodegenerative Diseases

(2015) M. Elizabeth Meredith et al.   AAPS Journal

Peptide therapeutics: current status and future directions

(2015) Keld Fosgerau et al.   DRUG DISCOVERY TODAY

Mextli proteins use both canonical bipartite and novel tripartite binding modes to form eIF4E complexes that display differential sensitivity to 4E-BP regulation

(2015) Daniel Peter et al.   GENES & DEVELOPMENT

MD and Docking Studies Reveal That the Functional Switch of CYFIP1 is Mediated by a Butterfly-like Motion

(2015) Daniele Di Marino et al.   Journal of Chemical Theory and Computation

PDE-4 Inhibition Rescues Aberrant Synaptic Plasticity in Drosophila and Mouse Models of Fragile X Syndrome

(2015) C. H. Choi et al.   JOURNAL OF NEUROSCIENCE

Targeting the translation machinery in cancer

(2015) Mamatha Bhat et al.   NATURE REVIEWS DRUG DISCOVERY

RNA-mediated epigenetic regulation of gene expression

(2015) Daniel Holoch et al.   NATURE REVIEWS GENETICS

Dysregulation and restoration of translational homeostasis in fragile X syndrome

(2015) Joel D. Richter et al.   NATURE REVIEWS NEUROSCIENCE

A 3′ untranslated region variant inFMR1eliminates neuronal activity-dependent translation of FMRP by disrupting binding of the RNA-binding protein HuR

(2015) Joshua A. Suhl et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A unique binding mode of the eukaryotic translation initiation factor 4E for guiding the design of novel peptide inhibitors

(2015) Daniele Di Marino et al.   PROTEIN SCIENCE

Exploring the role of MKK7 in excitotoxicity and cerebral ischemia: a novel pharmacological strategy against brain injury

(2015) A Vercelli et al.   Cell Death & Disease

Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons

(2015) Chul-Yong Park et al.   Cell Reports

Effect of lovastatin on behavior in children and adults with fragile X syndrome: An open-label study

(2014) Artuela Çaku et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Fragile X syndrome due to a missense mutation

(2014) Leila K Myrick et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Pharmacological rescue of Ras signaling, GluA1-dependent synaptic plasticity, and learning deficits in a fragile X model

(2014) C.-S. Lim et al.   GENES & DEVELOPMENT

Quantitative Phosphoproteomics of Murine Fmr1-KO Cell Lines Provides New Insights into FMRP-Dependent Signal Transduction Mechanisms

(2014) Katarina Matic et al.   JOURNAL OF PROTEOME RESEARCH

Activity-dependent alterations in the sensitivity to BDNF-TrkB signaling may promote excessive dendritic arborization and spinogenesis in fragile X syndrome in order to compensate for compromised postsynaptic activity

(2014) Sang Woo Kim et al.   MEDICAL HYPOTHESES

Reduced Phenotypic Severity Following Adeno-Associated Virus-Mediated Fmr1 Gene Delivery in Fragile X Mice

(2014) Shervin Gholizadeh et al.   NEUROPSYCHOPHARMACOLOGY

An eIF4E-interacting peptide induces cell death in cancer cell lines

(2014) M Masse et al.   Cell Death & Disease

4E-BPs require non-canonical 4E-binding motifs and a lateral surface of eIF4E to repress translation

(2014) Cátia Igreja et al.   Nature Communications

Pharmacogenetic Inhibition of eIF4E-Dependent Mmp9 mRNA Translation Reverses Fragile X Syndrome-like Phenotypes

(2014) Christos G. Gkogkas et al.   Cell Reports

Future directions for peptide therapeutics development

(2013) Allan A. Kaspar et al.   DRUG DISCOVERY TODAY

Comprehensive analysis of the transcriptional landscape of the human FMR1 gene reveals two new long noncoding RNAs differentially expressed in Fragile X syndrome and Fragile X-associated tremor/ataxia syndrome

(2013) Chiara Pastori et al.   HUMAN GENETICS

Molecular dynamics simulations show how the FMRP Ile304Asn mutation destabilizes the KH2 domain structure and affects its function

(2013) Daniele Di Marino et al.   JOURNAL OF BIOMOLECULAR STRUCTURE & DYNAMICS

A Randomized Double-Blind, Placebo-Controlled Trial of Minocycline in Children and Adolescents with Fragile X Syndrome

(2013) Mary Jacena S. Leigh et al.   JOURNAL OF DEVELOPMENTAL AND BEHAVIORAL PEDIATRICS

Targeting the endocannabinoid system in the treatment of fragile X syndrome

(2013) Arnau Busquets-Garcia et al.   NATURE MEDICINE

Lovastatin Corrects Excess Protein Synthesis and Prevents Epileptogenesis in a Mouse Model of Fragile X Syndrome

(2013) Emily K. Osterweil et al.   NEURON

Long-lasting effects of minocycline on behavior in young but not adult Fragile X mice

(2013) L.E. Dansie et al.   NEUROSCIENCE

Resilience to audiogenic seizures is associated with p-ERK1/2 dephosphorylation in the subiculum of Fmr1 knockout mice

(2013) Giulia Curia et al.   Frontiers in Cellular Neuroscience

Rational Optimization of Conformational Effects Induced By Hydrocarbon Staples in Peptides and their Binding Interfaces

(2013) Dilraj Lama et al.   Scientific Reports

The cap-translation inhibitor 4EGI-1 induces apoptosis in multiple myeloma through Noxa induction

(2012) G Descamps et al.   BRITISH JOURNAL OF CANCER

mTOR Signaling in Growth Control and Disease

(2012) Mathieu Laplante et al.   CELL

Fragile X syndrome: causes, diagnosis, mechanisms, and therapeutics

(2012) Claudia Bagni et al.   JOURNAL OF CLINICAL INVESTIGATION

Uncoupling of the endocannabinoid signalling complex in a mouse model of fragile X syndrome

(2012) Kwang-Mook Jung et al.   Nature Communications

Neural circuit architecture defects in a Drosophila model of Fragile X syndrome are alleviated by minocycline treatment and genetic removal of matrix metalloproteinase

(2011) S. S. Siller et al.   Disease Models & Mechanisms

Influence of matrix metalloproteinase MMP-9 on dendritic spine morphology

(2011) P. Michaluk et al.   JOURNAL OF CELL SCIENCE

Therapeutic Strategies in Fragile X Syndrome: Dysregulated mGluR Signaling and Beyond

(2011) Christina Gross et al.   NEUROPSYCHOPHARMACOLOGY

Cannabidiol Reduces the Anxiety Induced by Simulated Public Speaking in Treatment-Naïve Social Phobia Patients

(2011) Mateus M Bergamaschi et al.   NEUROPSYCHOPHARMACOLOGY

Chemical Modifications Designed to Improve Peptide Stability: Incorporation of Non-Natural Amino Acids, Pseudo-Peptide Bonds, and Cyclization

(2010) Luca Gentilucci et al.   CURRENT PHARMACEUTICAL DESIGN

Dysregulation of mTOR Signaling in Fragile X Syndrome

(2010) A. Sharma et al.   JOURNAL OF NEUROSCIENCE

Physiological Activation of Synaptic Rac>PAK (p-21 Activated Kinase) Signaling Is Defective in a Mouse Model of Fragile X Syndrome

(2010) L. Y. Chen et al.   JOURNAL OF NEUROSCIENCE

Structure and control of the actin regulatory WAVE complex

(2010) Zhucheng Chen et al.   NATURE

Abnormal mGlu 5 Receptor/Endocannabinoid Coupling in Mice Lacking FMRP and BC1 RNA

(2010) Mauro Maccarrone et al.   NEUROPSYCHOPHARMACOLOGY

Dendritic spine instability and insensitivity to modulation by sensory experience in a mouse model of fragile X syndrome

(2010) Feng Pan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Regulation of Translation Initiation in Eukaryotes: Mechanisms and Biological Targets

(2009) Nahum Sonenberg et al.   CELL

Inhibition of Ovarian Cancer Growth by a Tumor-Targeting Peptide That Binds Eukaryotic Translation Initiation Factor 4E

(2009) S. Y. Ko et al.   CLINICAL CANCER RESEARCH

Fragile X mental retardation protein replacement restores hippocampal synaptic function in a mouse model of fragile X syndrome

(2009) Z Zeier et al.   GENE THERAPY

Fragile X mental retardation protein control of neuronal mRNA metabolism: Insights into mRNA stability

(2009) Silvia De Rubeis et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

The Fragile X Syndrome Protein Represses Activity-Dependent Translation through CYFIP1, a New 4E-BP

(2008) Ilaria Napoli et al.   CELL

Downregulation of Tonic GABAergic Inhibition in a Mouse Model of Fragile X Syndrome

(2008) Giulia Curia et al.   CEREBRAL CORTEX

Actin in action: the interplay between the actin cytoskeleton and synaptic efficacy

(2008) Lorenzo A. Cingolani et al.   NATURE REVIEWS NEUROSCIENCE

Aberrant early-phase ERK inactivation impedes neuronal function in fragile X syndrome

(2008) S. H. Kim et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Abnormal Striatal GABA Transmission in the Mouse Model for the Fragile X Syndrome

(2007) Diego Centonze et al.   BIOLOGICAL PSYCHIATRY