Discovery of a biomarker and lead small molecules to target r(GGGGCC)-associated defects in c9FTD/ALS.
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From animal models to human disease: a genetic approach for personalized medicine in ALSThere has been an awakening: Emerging mechanisms of C9orf72 mutations in FTD/ALSClinical Trial Designs in Amyotrophic Lateral Sclerosis: Does One Design Fit All?Structure based approaches for targeting non-coding RNAs with small moleculesTDP-43 Proteinopathy and ALS: Insights into Disease Mechanisms and Therapeutic Targets.RNA-mediated pathogenic mechanisms in polyglutamine diseases and amyotrophic lateral sclerosisG-quadruplexes: Emerging roles in neurodegenerative diseases and the non-coding transcriptomeA comprehensive library of familial human amyotrophic lateral sclerosis induced pluripotent stem cellsApproaches to Validate and Manipulate RNA Targets with Small Molecules in CellsStructure and Dynamics of DNA and RNA Double Helices Obtained from the GGGGCC and CCCCGG Hexanucleotide Repeats That Are the Hallmark of C9FTD/ALS Diseases.RNA metabolism in neurodegenerative disease.A C9ORF72 BAC mouse model recapitulates key epigenetic perturbations of ALS/FTDPoly-GP in cerebrospinal fluid links C9orf72-associated dipeptide repeat expression to the asymptomatic phase of ALS/FTD.Frontotemporal lobar degeneration: defining phenotypic diversity through personalized medicine.Linking RNA Dysfunction and Neurodegeneration in Amyotrophic Lateral Sclerosis.The Spectrum of C9orf72-mediated Neurodegeneration and Amyotrophic Lateral Sclerosis.RAN translation at CGG repeats induces ubiquitin proteasome system impairment in models of fragile X-associated tremor ataxia syndromeRNA Structures as Mediators of Neurological Diseases and as Drug Targets.Cerebellar c9RAN proteins associate with clinical and neuropathological characteristics of C9ORF72 repeat expansion carriers.Distribution of dipeptide repeat proteins in cellular models and C9orf72 mutation cases suggests link to transcriptional silencing(Patho-)physiological relevance of PINK1-dependent ubiquitin phosphorylationDifferential Toxicity of Nuclear RNA Foci versus Dipeptide Repeat Proteins in a Drosophila Model of C9ORF72 FTD/ALS.Distinct C9orf72-Associated Dipeptide Repeat Structures Correlate with Neuronal Toxicity.Novel clinical associations with specific C9ORF72 transcripts in patients with repeat expansions in C9ORF72.Defining RNA-Small Molecule Affinity Landscapes Enables Design of a Small Molecule Inhibitor of an Oncogenic Noncoding RNAC9orf72 BAC Transgenic Mice Display Typical Pathologic Features of ALS/FTDNeurodegeneration. C9ORF72 repeat expansions in mice cause TDP-43 pathology, neuronal loss, and behavioral deficits.ALS biomarkers for therapy development: State of the field and future directions.Retention of hexanucleotide repeat-containing intron in C9orf72 mRNA: implications for the pathogenesis of ALS/FTDUse of biomarkers in ALS drug development and clinical trialsHuman C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic MiceGain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAsInforna 2.0: A Platform for the Sequence-Based Design of Small Molecules Targeting Structured RNAsTreatment of frontotemporal dementia.Poly-dipeptides encoded by the C9ORF72 repeats block global protein translationLongitudinal imaging in C9orf72 mutation carriers: Relationship to phenotype.Drosophila melanogaster As a Model Organism to Study RNA Toxicity of Repeat Expansion-Associated Neurodegenerative and Neuromuscular DiseasesReduction of Huntington's Disease RNA Foci by CAG Repeat-Targeting Reagents.Development of Therapeutics for C9ORF72 ALS/FTD-Related Disorders.Compromised MAPK signaling in human diseases: an update.
P2860
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P2860
Discovery of a biomarker and lead small molecules to target r(GGGGCC)-associated defects in c9FTD/ALS.
description
2014 nî lūn-bûn
@nan
2014 թուականի Օգոստոսին հրատարակուած գիտական յօդուած
@hyw
2014 թվականի օգոստոսին հրատարակված գիտական հոդված
@hy
2014年の論文
@ja
2014年論文
@yue
2014年論文
@zh-hant
2014年論文
@zh-hk
2014年論文
@zh-mo
2014年論文
@zh-tw
2014年论文
@wuu
name
Discovery of a biomarker and lead small molecules to target r
@nl
Discovery of a biomarker and l ...... sociated defects in c9FTD/ALS.
@ast
Discovery of a biomarker and l ...... sociated defects in c9FTD/ALS.
@en
type
label
Discovery of a biomarker and lead small molecules to target r
@nl
Discovery of a biomarker and l ...... sociated defects in c9FTD/ALS.
@ast
Discovery of a biomarker and l ...... sociated defects in c9FTD/ALS.
@en
prefLabel
Discovery of a biomarker and lead small molecules to target r
@nl
Discovery of a biomarker and l ...... sociated defects in c9FTD/ALS.
@ast
Discovery of a biomarker and l ...... sociated defects in c9FTD/ALS.
@en
P2093
P2860
P50
P1433
P1476
Discovery of a biomarker and l ...... sociated defects in c9FTD/ALS.
@en
P2093
Amelia Johnston
Antonia Ratti
Bradley F Boeve
Bryan J Traynor
Claudia Morelli
Erik Fostvedt
James D Berry
Jeannie Chew
Jeffrey D Rothstein
Karen Jansen-West
P2860
P304
P356
10.1016/J.NEURON.2014.07.041
P407
P50
P577
2014-08-14T00:00:00Z