Exploring the Potential of Small Molecule-Based Therapeutic Approaches for Targeting Trinucleotide Repeat Disorders
出版年份 2019 全文链接
标题
Exploring the Potential of Small Molecule-Based Therapeutic Approaches for Targeting Trinucleotide Repeat Disorders
作者
关键词
Trinucleotide repeats, Small molecules, RNA, Therapeutics, Neurological disorders
出版物
MOLECULAR NEUROBIOLOGY
Volume -, Issue -, Pages -
出版商
Springer Science and Business Media LLC
发表日期
2019-08-10
DOI
10.1007/s12035-019-01724-4
参考文献
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注意：仅列出部分参考文献，下载原文获取全部文献信息。- Development of novel macrocyclic small molecules that target CTG trinucleotide repeats
- (2019) Julio F. Serrano et al. BIOORGANIC & MEDICINAL CHEMISTRY
- Precise small-molecule cleavage of an r(CUG) repeat expansion in a myotonic dystrophy mouse model
- (2019) Alicia J. Angelbello et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Piperine Modulates Protein Mediated Toxicity in Fragile X-Associated Tremor/Ataxia Syndrome through Interacting Expanded CGG Repeat (r(CGG)exp) RNA
- (2019) Arun Kumar Verma et al. ACS Chemical Neuroscience
- Rationally designed small molecules targeting toxic CAG repeat RNA that causes Huntington's disease (HD) and spinocerebellar ataxia (SCAs)
- (2019) Eshan Khan et al. BIOCHIMIE
- Reducing Mutant Huntingtin Protein Expression in Living Cells by a Newly Identified RNA CAG Binder
- (2018) Frank Matthes et al. ACS Chemical Neuroscience
- Targeting the CACNA1A IRES as a Treatment for Spinocerebellar Ataxia Type 6
- (2018) Parviz Daniel Hejazi Pastor et al. CEREBELLUM
- Bicyclic and tricyclic C–C mismatch-binding ligands bind to CCG trinucleotide repeat DNAs
- (2018) Tomonori Shibata et al. CHEMICAL COMMUNICATIONS
- Piperine ameliorates SCA17 neuropathology by reducing ER stress
- (2018) Jifeng Guo et al. Molecular Neurodegeneration
- The indole compound NC009-1 inhibits aggregation and promotes neurite outgrowth through enhancement of HSPB1 in SCA17 cells and ameliorates the behavioral deficits in SCA17 mice
- (2018) Chiung-Mei Chen et al. NEUROTOXICOLOGY
- Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat
- (2018) Liande Li et al. Nucleic Acid Therapeutics
- Myotonic Dystrophies: Targeting Therapies for Multisystem Disease
- (2018) Samantha LoRusso et al. Neurotherapeutics
- Temporomandibular dysfunction in adult patients with myotonic dystrophy (DM1)
- (2017) C. Mejersjö et al. JOURNAL OF ORAL REHABILITATION
- Synthetic transcription elongation factors license transcription across repressive chromatin
- (2017) Graham S. Erwin et al. SCIENCE
- Synthesis and characterization of a bifunctional nanoprobe for CGG trinucleotide repeat detection
- (2017) Xiaoqian Zhu et al. RSC Advances
- Small Molecule Recognition and Tools to Study Modulation of r(CGG)exp in Fragile X-Associated Tremor Ataxia Syndrome
- (2016) Wang-Yong Yang et al. ACS Chemical Biology
- Synthesis of 1H-pyrrolo[3,2-h]quinoline-8-amine derivatives that target CTG trinucleotide repeats
- (2016) Jun Matsumoto et al. BIOORGANIC & MEDICINAL CHEMISTRY LETTERS
- Naphthyridine-Benzoazaquinolone: Evaluation of a Tricyclic System for the Binding to (CAG)nRepeat DNA and RNA
- (2016) Jinxing Li et al. Chemistry-An Asian Journal
- A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in aDrosophilaModel of Myotonic Dystrophy
- (2016) Long M. Luu et al. ChemMedChem
- Sustained expression ofFMR1mRNA from reactivated fragile X syndrome alleles after treatment with small molecules that prevent trimethylation of H3K27
- (2016) Daman Kumari et al. HUMAN MOLECULAR GENETICS
- Precise small-molecule recognition of a toxic CUG RNA repeat expansion
- (2016) Suzanne G Rzuczek et al. Nature Chemical Biology
- The P42 peptide and Peptide-based therapies for Huntington’s disease
- (2016) Cecilia Marelli et al. Orphanet Journal of Rare Diseases
- Alleviating GAA Repeat Induced Transcriptional Silencing of the Friedreich's Ataxia Gene During Somatic Cell Reprogramming
- (2016) Urszula Polak et al. STEM CELLS AND DEVELOPMENT
- Treatment of fragile X-associated tremor ataxia syndrome (FXTAS) and related neurological problems
- (2016) Randi Hagerman Clinical Interventions in Aging
- A Huntingtin-based peptide inhibitor of caspase-6 provides protection from mutant Huntingtin-induced motor and behavioral deficits
- (2015) Israel Aharony et al. HUMAN MOLECULAR GENETICS
- Riluzole in patients with hereditary cerebellar ataxia: a randomised, double-blind, placebo-controlled trial
- (2015) Silvia Romano et al. LANCET NEUROLOGY
- Structural Insights Reveal the Dynamics of the Repeating r(CAG) Transcript Found in Huntington’s Disease (HD) and Spinocerebellar Ataxias (SCAs)
- (2015) Arpita Tawani et al. PLoS One
- Triptycene-based small molecules modulate (CAG)·(CTG) repeat junctions
- (2015) Stephanie A. Barros et al. Chemical Science
- Actinomycin D Specifically Reduces Expanded CUG Repeat RNA in Myotonic Dystrophy Models
- (2015) Ruth B. Siboni et al. Cell Reports
- Targeting the r(CGG) Repeats That Cause FXTAS with Modularly Assembled Small Molecules and Oligonucleotides
- (2014) Tuan Tran et al. ACS Chemical Biology
- Epigenetic therapy for Friedreich ataxia
- (2014) Elisabetta Soragni et al. ANNALS OF NEUROLOGY
- Allele-Selective Inhibition of Mutant Atrophin-1 Expression by Duplex and Single-Stranded RNAs
- (2014) Jiaxin Hu et al. BIOCHEMISTRY
- Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts
- (2014) Janine Scholefield et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Dyclonine rescues frataxin deficiency in animal models and buccal cells of patients with Friedreich's ataxia
- (2014) Sunil Sahdeo et al. HUMAN MOLECULAR GENETICS
- A randomized controlled pilot trial of lithium in spinocerebellar ataxia type 2
- (2014) Francesco Saccà et al. JOURNAL OF NEUROLOGY
- SCA 17 phenotype with intermediate triplet repeat number
- (2014) Heather Herrema et al. JOURNAL OF THE NEUROLOGICAL SCIENCES
- Epigenetic and neurological effects and safety of high-dose nicotinamide in patients with Friedreich's ataxia: an exploratory, open-label, dose-escalation study
- (2014) Vincenzo Libri et al. LANCET
- Nonallele Specific Silencing of Ataxin-7 Improves Disease Phenotypes in a Mouse Model of SCA7
- (2014) Pavitra S Ramachandran et al. MOLECULAR THERAPY
- Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia
- (2014) Morgane Perdomini et al. NATURE MEDICINE
- Myotonic Dystrophy
- (2014) Charles A. Thornton NEUROLOGIC CLINICS
- Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects
- (2014) Jason W. Hoskins et al. NUCLEIC ACIDS RESEARCH
- Indole and synthetic derivative activate chaperone expression to reduce polyQ aggregation in SCA17 neuronal cell and slice culture models
- (2014) Guey-Jen Lee-Chen et al. Drug Design Development and Therapy
- Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy
- (2014) Andrew P. Lieberman et al. Cell Reports
- R-loops Associated with Triplet Repeat Expansions Promote Gene Silencing in Friedreich Ataxia and Fragile X Syndrome
- (2014) Matthias Groh et al. PLoS Genetics
- Reducing Levels of Toxic RNA with Small Molecules
- (2013) Leslie A. Coonrod et al. ACS Chemical Biology
- Large normal alleles and SCA2 prevalence: lessons from a nationwide study and analysis of the literature
- (2013) J.M. Laffita-Mesa et al. CLINICAL GENETICS
- Heterochromatinization induced by GAA-repeat hyperexpansion in Friedreich's ataxia can be reduced upon HDAC inhibition by vitamin B3
- (2013) P. K. Chan et al. HUMAN MOLECULAR GENETICS
- Prevalence of adult Huntington's disease in the UK based on diagnoses recorded in general practice records
- (2013) Stephen JW Evans et al. JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY
- Congenital Myotonic Dystrophy: Canadian Population-Based Surveillance Study
- (2013) Craig Campbell et al. JOURNAL OF PEDIATRICS
- A Dynamic Structural Model of Expanded RNA CAG Repeats: A Refined X-ray Structure and Computational Investigations Using Molecular Dynamics and Umbrella Sampling Simulations
- (2013) Ilyas Yildirim et al. JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
- The pridopidine paradox in Huntington's disease
- (2013) Ralf Reilmann MOVEMENT DISORDERS
- A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease
- (2013) MOVEMENT DISORDERS
- A Small Molecule That Targets r(CGG)exp and Improves Defects in Fragile X-Associated Tremor Ataxia Syndrome
- (2012) Matthew D. Disney et al. ACS Chemical Biology
- Chemical Correction of Pre-mRNA Splicing Defects Associated with Sequestration of Muscleblind-like 1 Protein by Expanded r(CAG)-Containing Transcripts
- (2012) Amit Kumar et al. ACS Chemical Biology
- Brain pathology of spinocerebellar ataxias
- (2012) Kay Seidel et al. ACTA NEUROPATHOLOGICA
- Multivalency as a Chemical Organization and Action Principle
- (2012) Carlo Fasting et al. ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
- A small molecule regulates hairpin structures in d(CGG) trinucleotide repeats
- (2012) Masaki Hagihara et al. BIOORGANIC & MEDICINAL CHEMISTRY LETTERS
- Severe and rapidly progressing cognitive phenotype in a SCA17-family with only marginally expanded CAG/CAA repeats in the TATA-box binding protein gene: A case report
- (2012) Troels Tolstrup Nielsen et al. BMC Neurology
- Lisuride Reduces Involuntary Periodic Leg Movements in Spinocerebellar Ataxia Type 2 Patients
- (2012) Luis Velázquez-Pérez et al. CEREBELLUM
- Chemical screen reveals small molecules suppressing fragile X premutation rCGG repeat-mediated neurodegeneration in Drosophila
- (2012) A. Qurashi et al. HUMAN MOLECULAR GENETICS
- Spinocerebellar Ataxia Type 7: Report of a New Italian Family
- (2012) Domenico Italiano et al. INTERNAL MEDICINE
- Clinical Features of Friedreich Ataxia
- (2012) Martin B. Delatycki et al. JOURNAL OF CHILD NEUROLOGY
- Fragile X syndrome: causes, diagnosis, mechanisms, and therapeutics
- (2012) Claudia Bagni et al. JOURNAL OF CLINICAL INVESTIGATION
- Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
- (2012) Holly B. Kordasiewicz et al. NEURON
- Fragile X syndrome
- (2011) Jeremy Turk CURRENT OPINION IN PSYCHIATRY
- Population frequency of myotonic dystrophy: higher than expected frequency of myotonic dystrophy type 2 (DM2) mutation in Finland
- (2011) Tiina Suominen et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Unexpanded and intermediate CAG polymorphisms at the SCA2 locus (ATXN2) in the Cuban population: evidence about the origin of expanded SCA2 alleles
- (2011) José Miguel Laffita-Mesa et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Mortality in Friedreich Ataxia
- (2011) Amy Y. Tsou et al. JOURNAL OF THE NEUROLOGICAL SCIENCES
- Pridopidine for the treatment of motor function in patients with Huntington's disease (MermaiHD): a phase 3, randomised, double-blind, placebo-controlled trial
- (2011) Justo Garcia de Yebenes et al. LANCET NEUROLOGY
- Efficacy and safety of dutasteride in patients with spinal and bulbar muscular atrophy: a randomised placebo-controlled trial
- (2011) Lindsay E Fernández-Rhodes et al. LANCET NEUROLOGY
- Prolonged treatment with pimelic o-aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model
- (2011) Chiranjeevi Sandi et al. NEUROBIOLOGY OF DISEASE
- Oral Zinc Sulphate Supplementation for Six Months in SCA2 Patients: A Randomized, Double-Blind, Placebo-Controlled Trial
- (2011) Luis Velázquez-Pérez et al. NEUROCHEMICAL RESEARCH
- Triplet repeat RNA structure and its role as pathogenic agent and therapeutic target
- (2011) W. J. Krzyzosiak et al. NUCLEIC ACIDS RESEARCH
- Efficacy and Safety of the Dopaminergic Stabilizer Pridopidine (ACR16) in Patients With Huntington's Disease
- (2010) Anders Lundin et al. CLINICAL NEUROPHARMACOLOGY
- Efficacy and safety of leuprorelin in patients with spinal and bulbar muscular atrophy (JASMITT study): a multicentre, randomised, double-blind, placebo-controlled trial
- (2010) Masahisa Katsuno et al. LANCET NEUROLOGY
- Ataxin-2 intermediate-length polyglutamine expansions are associated with increased risk for ALS
- (2010) Andrew C. Elden et al. NATURE
- Repeat instability as the basis for human diseases and as a potential target for therapy
- (2010) Arturo López Castel et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- The carboxy-terminal fragment of α1A calcium channel preferentially aggregates in the cytoplasm of human spinocerebellar ataxia type 6 Purkinje cells
- (2009) Taro Ishiguro et al. ACTA NEUROPATHOLOGICA
- Phase 2 trial of leuprorelin in patients with spinal and bulbar muscular atrophy
- (2009) Haruhiko Banno et al. ANNALS OF NEUROLOGY
- A Small Molecule Affecting the Replication of Trinucleotide Repeat d(GAA)n
- (2009) Hanping He et al. CHEMISTRY-A EUROPEAN JOURNAL
- Treatments for fragile X syndrome: A closer look at the data
- (2009) Scott S. Hall Developmental Disabilities Research Reviews
- Prevention of oculopharyngeal muscular dystrophy by muscular expression of Llama single-chain intrabodies in vivo
- (2009) Aymeric Chartier et al. HUMAN MOLECULAR GENETICS
- Screening for Expanded Alleles of the FMR1 Gene in Blood Spots from Newborn Males in a Spanish Population
- (2009) Isabel Fernandez-Carvajal et al. JOURNAL OF MOLECULAR DIAGNOSTICS
- Friedreich ataxia: The clinical picture
- (2009) Massimo Pandolfo JOURNAL OF NEUROLOGY
- Decreased androgen concentrations and diminished general and sexual well-being in women with premature ovarian failure
- (2009) Jolande G. van der Stege et al. MENOPAUSE-THE JOURNAL OF THE NORTH AMERICAN MENOPAUSE SOCIETY
- Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs
- (2009) Jiaxin Hu et al. NATURE BIOTECHNOLOGY
- Molecular epidemiology of spinocerebellar ataxias in Cuba: Insights into SCA2 founder effect in Holguin
- (2009) Luis Velázquez Pérez et al. NEUROSCIENCE LETTERS
- Design of RNAi Hairpins for Mutation-Specific Silencing of Ataxin-7 and Correction of a SCA7 Phenotype
- (2009) Janine Scholefield et al. PLoS One
- Pentamidine reverses the splicing defects associated with myotonic dystrophy
- (2009) M. B. Warf et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Treatment of imbalance with varenicline (Chantix®): report of a patient with fragile X tremor/ataxia syndrome
- (2008) T. A. Zesiewicz et al. ACTA NEUROLOGICA SCANDINAVICA
- Expanded clinical phenotype of women with theFMR1 premutation
- (2008) Sarah M. Coffey et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Sporadic ataxias in Japan – a population-based epidemiological study
- (2008) Shoji Tsuji et al. CEREBELLUM
- Treatment of Ataxia and Imbalance With Varenicline (Chantix)
- (2008) Theresa A. Zesiewicz et al. CLINICAL NEUROPHARMACOLOGY
- Advances in the Treatment of Fragile X Syndrome
- (2008) R. J. Hagerman et al. PEDIATRICS
- Spinocerebellar ataxia type 7 (SCA7): widespread brain damage in an adult-onset patient with progressive visual impairments in comparison with an adult-onset patient without visual impairments
- (2007) U. Rüb et al. NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY
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