Therapeutics development for triplet repeat expansion diseases (Q36277195)

13 August 2017

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title

Therapeutics development for triplet repeat expansion diseases (English)

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13 August 2017

Nicholas A Di Prospero

series ordinal

1

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PubMed ID

16205715

13 August 2017

Kenneth H Fischbeck

series ordinal

2

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13 August 2017

1 October 2005

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13 August 2017

Nature Reviews Genetics

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13 August 2017

6

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13 August 2017

10

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13 August 2017

756-765

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https://scigraph.springernature.com/pub.10.1038/nrg1690

13 August 2017

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cites work

Androgen receptor gene mutations in X-linked spinal and bulbar muscular atrophy

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Fourteen and counting: unraveling trinucleotide repeat diseases

21 January 2018

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Frataxin is reduced in Friedreich ataxia patients and is associated with mitochondrial membranes

21 January 2018

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Frataxin and frataxin deficiency in Friedreich's ataxia

21 January 2018

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Molecular basis of Friedreich ataxia

21 January 2018

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Idebenone. A review of its pharmacodynamic and pharmacokinetic properties, and therapeutic use in age-related cognitive disorders

21 January 2018

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Molecular insights into Friedreich's ataxia and antioxidant-based therapies

21 January 2018

1 reference

Friedreich's ataxia: idebenone treatment in early stage patients

21 January 2018

1 reference

Selective targeting of a redox-active ubiquinone to mitochondria within cells: antioxidant and antiapoptotic properties

21 January 2018

1 reference

A study of intracellular iron metabolism using pyridoxal isonicotinoyl hydrazone and other synthetic chelating agents

21 January 2018

1 reference

Development of potential iron chelators for the treatment of Friedreich's ataxia: ligands that mobilize mitochondrial iron

21 January 2018

1 reference

Upregulation of expression from the FRDA genomic locus for the therapy of Friedreich ataxia

21 January 2018

1 reference

Cisplatin may induce frataxin expression

21 January 2018

1 reference

Absence of expression of the FMR-1 gene in fragile X syndrome

21 January 2018

1 reference

In vitro reactivation of the FMR1 gene involved in fragile X syndrome

21 January 2018

1 reference

Modulation of methylation in the FMR1 promoter region after long term treatment with L-carnitine and acetyl-L-carnitine

21 January 2018

1 reference

Acetylated histones are associated with FMR1 in normal but not fragile X-syndrome cells.

21 January 2018

1 reference

Synergistic effect of histone hyperacetylation and DNA demethylation in the reactivation of the FMR1 gene.

21 January 2018

1 reference

Dendritic spine and dendritic field characteristics of layer V pyramidal neurons in the visual cortex of fragile-X knockout mice.

21 January 2018

1 reference

Altered synaptic plasticity in a mouse model of fragile X mental retardation

21 January 2018

1 reference

The mGluR theory of fragile X mental retardation

21 January 2018

1 reference

Pharmacological rescue of synaptic plasticity, courtship behavior, and mushroom body defects in a Drosophila model of fragile X syndrome

21 January 2018

1 reference

An unstable triplet repeat in a gene related to myotonic muscular dystrophy

21 January 2018

1 reference

RNA CUG repeats sequester CUGBP1 and alter protein levels and activity of CUGBP1

21 January 2018

1 reference

Ribozyme-mediated trans-splicing of a trinucleotide repeat.

21 January 2018

1 reference

Some flavonoids and DHEA-S prevent the cis-effect of expanded CTG repeats in a stable PC12 cell transformant.

21 January 2018

1 reference

Small molecule activators of sirtuins extend Saccharomyces cerevisiae lifespan

21 January 2018

1 reference

Resveratrol rescues mutant polyglutamine cytotoxicity in nematode and mammalian neurons.

21 January 2018

1 reference

Behavioural abnormalities and selective neuronal loss in HD transgenic mice expressing mutated full-length HD cDNA.

21 January 2018

1 reference

Transgenic mice harboring a full-length human mutant DRPLA gene exhibit age-dependent intergenerational and somatic instabilities of CAG repeats comparable with those in DRPLA patients.

21 January 2018

1 reference

Testosterone reduction prevents phenotypic expression in a transgenic mouse model of spinal and bulbar muscular atrophy

21 January 2018

1 reference

Wild-type huntingtin plays a role in brain development and neuronal survival

21 January 2018

1 reference

Chaperone suppression of aggregation and altered subcellular proteasome localization imply protein misfolding in SCA1.

21 January 2018

1 reference

Evidence for proteasome involvement in polyglutamine disease: localization to nuclear inclusions in SCA3/MJD and suppression of polyglutamine aggregation in vitro

21 January 2018

1 reference

Genetic suppression of polyglutamine toxicity in Drosophila

21 January 2018

1 reference

Effects of heat shock, heat shock protein 40 (HDJ-2), and proteasome inhibition on protein aggregation in cellular models of Huntington's disease

21 January 2018

1 reference

Geldanamycin activates a heat shock response and inhibits huntingtin aggregation in a cell culture model of Huntington's disease

21 January 2018

1 reference

Identification of benzothiazoles as potential polyglutamine aggregation inhibitors of Huntington's disease by using an automated filter retardation assay

21 January 2018

1 reference

Inhibition of polyglutamine aggregation in R6/2 HD brain slices-complex dose-response profiles

21 January 2018

1 reference

Pivotal role of oligomerization in expanded polyglutamine neurodegenerative disorders.

21 January 2018

1 reference

Trehalose alleviates polyglutamine-mediated pathology in a mouse model of Huntington disease

21 January 2018

1 reference

Effects of intracellular expression of anti-huntingtin antibodies of various specificities on mutant huntingtin aggregation and toxicity

21 January 2018

1 reference

A bivalent Huntingtin binding peptide suppresses polyglutamine aggregation and pathogenesis in Drosophila

21 January 2018

1 reference

Peptides containing glutamine repeats as substrates for transglutaminase-catalyzed cross-linking: relevance to diseases of the nervous system

21 January 2018

1 reference

Transglutaminase aggregates huntingtin into nonamyloidogenic polymers, and its enzymatic activity increases in Huntington's disease brain nuclei

21 January 2018

1 reference

Prolonged survival and decreased abnormal movements in transgenic model of Huntington disease, with administration of the transglutaminase inhibitor cystamine

21 January 2018

1 reference

Cystamine inhibits caspase activity. Implications for the treatment of polyglutamine disorders

21 January 2018

1 reference

Tissue transglutaminase contributes to disease progression in the R6/2 Huntington's disease mouse model via aggregate-independent mechanisms

21 January 2018

1 reference

Caspases and neurodegeneration: on the cutting edge of new therapeutic approaches.

21 January 2018

1 reference

Cleavage of huntingtin by apopain, a proapoptotic cysteine protease, is modulated by the polyglutamine tract

21 January 2018

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Caspase cleavage of gene products associated with triplet expansion disorders generates truncated fragments containing the polyglutamine tract

21 January 2018

1 reference

Inhibiting caspase cleavage of huntingtin reduces toxicity and aggregate formation in neuronal and nonneuronal cells

21 January 2018

1 reference

Inhibition of caspase-1 slows disease progression in a mouse model of Huntington's disease

21 January 2018

1 reference

Minocycline inhibits caspase-1 and caspase-3 expression and delays mortality in a transgenic mouse model of Huntington disease

21 January 2018

1 reference

Minocycline inhibits caspase-independent and -dependent mitochondrial cell death pathways in models of Huntington's disease

21 January 2018

1 reference

Neuroprotection in Huntington's disease: a 2-year study on minocycline

21 January 2018

1 reference

Tauroursodeoxycholic acid, a bile acid, is neuroprotective in a transgenic animal model of Huntington's disease.

21 January 2018

1 reference

Lesion of striatal neurons with kainic acid provides a model for Huntington's chorea

21 January 2018

1 reference

Replication of the neurochemical characteristics of Huntington's disease by quinolinic acid

21 January 2018

1 reference

NMDA receptor function in mouse models of Huntington disease

21 January 2018

1 reference

Increased sensitivity to N-methyl-D-aspartate receptor-mediated excitotoxicity in a mouse model of Huntington's disease

21 January 2018

1 reference

Altered striatal amino acid neurotransmitter release monitored using microdialysis in R6/1 Huntington transgenic mice

21 January 2018

1 reference

Huntington's disease: a synaptopathy?

21 January 2018

1 reference

Altered neurotransmitter receptor expression in transgenic mouse models of Huntington's disease

21 January 2018

1 reference

A randomized trial of amantadine in Huntington disease

21 January 2018

1 reference

Amantadine in Huntington's disease: open-label video-blinded study

21 January 2018

1 reference

Huntington's disease: a randomized, controlled trial using the NMDA-antagonist amantadine

21 January 2018

1 reference

Cognitive, behavioral, and motor effects of the NMDA antagonist ketamine in Huntington's disease

21 January 2018

1 reference

A controlled clinical trial of baclofen as protective therapy in early Huntington's disease

21 January 2018

1 reference

Influence of lamotrigine on progression of early Huntington disease: a randomized clinical trial

21 January 2018

1 reference

Riluzole in Huntington's disease (HD): an open label study with one year follow up.

21 January 2018

1 reference

Role of NR2B-type NMDA receptors in selective neurodegeneration in Huntington disease

21 January 2018

1 reference

Enhanced striatal NR2B-containing N-methyl-D-aspartate receptor-mediated synaptic currents in a mouse model of Huntington disease

21 January 2018

1 reference

NR2A and NR2B receptor gene variations modify age at onset in Huntington disease

21 January 2018

1 reference

Mechanism of memantine block of NMDA-activated channels in rat retinal ganglion cells: uncompetitive antagonism

21 January 2018

1 reference

Group-I metabotropic glutamate receptors: hypotheses to explain their dual role in neurotoxicity and neuroprotection.

21 January 2018

1 reference

The metabotropic glutamate receptor 5 antagonist MPEP and the mGluR2 agonist LY379268 modify disease progression in a transgenic mouse model of Huntington's disease

21 January 2018

1 reference

Effects of cannabinoids in the rat model of Huntington's disease generated by an intrastriatal injection of malonate

21 January 2018

1 reference

Systemic 3-nitropropionic acid: behavioral deficits and striatal damage in adult rats

21 January 2018

1 reference

Mitochondrial defect in Huntington's disease caudate nucleus

21 January 2018

1 reference

Oxidative damage and metabolic dysfunction in Huntington's disease: selective vulnerability of the basal ganglia.

21 January 2018

1 reference

Experimental therapeutics in transgenic mouse models of Huntington's disease

21 January 2018

1 reference

Creatine therapy provides neuroprotection after onset of clinical symptoms in Huntington's disease transgenic mice

21 January 2018

1 reference

Creatine supplementation in Huntington's disease: a placebo-controlled pilot trial

21 January 2018

1 reference

A controlled trial of idebenone in Huntington's disease

21 January 2018

1 reference

Polyglutamine diseases: a transcription disorder?

21 January 2018

1 reference

CREB-binding protein sequestration by expanded polyglutamine

21 January 2018

1 reference

Sp1 and TAFII130 transcriptional activity disrupted in early Huntington's disease

21 January 2018

1 reference

Aberrant histone acetylation, altered transcription, and retinal degeneration in a Drosophila model of polyglutamine disease are rescued by CREB-binding protein

21 January 2018

1 reference

Heterochromatin and epigenetic control of gene expression

21 January 2018

1 reference

Histone deacetylase inhibitors arrest polyglutamine-dependent neurodegeneration in Drosophila

21 January 2018

1 reference

Inducible PC12 cell model of Huntington's disease shows toxicity and decreased histone acetylation

21 January 2018

1 reference

Suberoylanilide hydroxamic acid, a histone deacetylase inhibitor, ameliorates motor deficits in a mouse model of Huntington's disease

21 January 2018

1 reference

Long-term treatment with sodium phenylbutyrate in ornithine transcarbamylase-deficient patients

21 January 2018

1 reference

Histone deacetylase inhibitors: from target to clinical trials

21 January 2018

1 reference

Stress-dependent regulation of FOXO transcription factors by the SIRT1 deacetylase

21 January 2018

1 reference

Implants of polymer-encapsulated human NGF-secreting cells in the nonhuman primate: rescue and sprouting of degenerating cholinergic basal forebrain neurons

21 January 2018

1 reference

Brain-derived neurotrophic factor-mediated protection of striatal neurons in an excitotoxic rat model of Huntington's disease, as demonstrated by adenoviral gene transfer

21 January 2018

1 reference

Brain-derived neurotrophic factor, neurotrophin-3, and neurotrophin-4/5 prevent the death of striatal projection neurons in a rodent model of Huntington's disease

21 January 2018

1 reference

Structural and functional neuroprotection in a rat model of Huntington's disease by viral gene transfer of GDNF.

21 January 2018

1 reference

AAV-mediated gene delivery of BDNF or GDNF is neuroprotective in a model of Huntington disease

21 January 2018

1 reference

Brain-derived neurotrophic factor regulates the onset and severity of motor dysfunction associated with enkephalinergic neuronal degeneration in Huntington's disease.

21 January 2018

1 reference

Increased GABAergic function in mouse models of Huntington's disease: reversal by BDNF.

21 January 2018

1 reference

Lentiviral gene delivery of GDNF into the striatum of R6/2 Huntington mice fails to attenuate behavioral and neuropathological changes

21 January 2018

1 reference

Loss of huntingtin-mediated BDNF gene transcription in Huntington's disease

21 January 2018

1 reference

Androgen receptor YAC transgenic mice recapitulate SBMA motor neuronopathy and implicate VEGF164 in the motor neuron degeneration

21 January 2018

1 reference

Neuroprotective gene therapy for Huntington's disease, using polymer-encapsulated cells engineered to secrete human ciliary neurotrophic factor: results of a phase I study.

21 January 2018

1 reference

Striatal transplantation in a transgenic mouse model of Huntington's disease

21 January 2018

1 reference

Anterior cingulate cortical transplantation in transgenic Huntington's disease mice

21 January 2018

1 reference

Transplanted fetal striatum in Huntington's disease: phenotypic development and lack of pathology

21 January 2018

1 reference

Motor and cognitive improvements in patients with Huntington's disease after neural transplantation

21 January 2018

1 reference

Striatal neural grafting improves cortical metabolism in Huntington's disease patients

21 January 2018

1 reference

Human neural stem cell transplants improve motor function in a rat model of Huntington's disease

21 January 2018

1 reference

Autologous adult bone marrow stem cell transplantation in an animal model of huntington's disease: behavioral and morphological outcomes

21 January 2018

1 reference

Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease

21 January 2018

1 reference

Rescue of polyglutamine-mediated cytotoxicity by double-stranded RNA-mediated RNA interference

21 January 2018

1 reference

RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.

21 January 2018

1 reference

RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model

21 January 2018

1 reference

Neuroprotective agents for clinical trials in Parkinson's disease: a systematic assessment

21 January 2018

1 reference

Sustained efficacy and safety of idebenone in the treatment of Alzheimer's disease: update on a 2-year double-blind multicentre study

21 January 2018

1 reference

Mitochondria-targeted antioxidants protect Friedreich Ataxia fibroblasts from endogenous oxidative stress more effectively than untargeted antioxidants.

31 March 2020

1 reference

Fenton chemistry: an introduction.

31 March 2020

1 reference

Abnormal development of dendritic spines in FMR1 knock-out mice

31 March 2020

1 reference

Polyphenols: antioxidants and beyond

31 March 2020

1 reference

Therapeutic effects of cystamine in a murine model of Huntington's disease.

31 March 2020

1 reference

Epidermal immunization by a needle-free powder delivery technology: Immunogenicity of influenza vaccine and protection in mice

31 March 2020

1 reference

Blockade of striatal adenosine A2A receptor reduces, through a presynaptic mechanism, quinolinic acid-induced excitotoxicity: possible relevance to neuroprotective interventions in neurodegenerative diseases of the striatum.

31 March 2020

1 reference

Histone Deacetylase Inhibition by Sodium Butyrate Chemotherapy Ameliorates the Neurodegenerative Phenotype in Huntington's Disease Mice

31 March 2020

1 reference

Implants of encapsulated human CNTF-producing fibroblasts prevent behavioral deficits and striatal degeneration in a rodent model of Huntington's disease

31 March 2020

1 reference

Treatment in myotonia and periodic paralysis

31 March 2020

1 reference

inferred from DOI database lookup

7 January 2021

based on heuristic

A randomized, placebo-controlled trial of coenzyme Q10 and remacemide in Huntington's disease

1 reference

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1038/NRG1690

1 reference

13 August 2017

1 reference

PubMed ID

16205715