Mesenchymal Stromal Cells’ Therapy for Polyglutamine Disorders: Where Do We Stand and Where Should We Go? [PDF]
Frontiers in Cellular Neuroscience, 2020 Polyglutamine (polyQ) diseases are a group of inherited neurodegenerative disorders caused by the expansion of the cytosine-adenine-guanine (CAG) repeat.
Inês Barros +18 more
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Involvement of HDAC1 and HDAC3 in the Pathology of Polyglutamine Disorders: Therapeutic Implications for Selective HDAC1/HDAC3 Inhibitors [PDF]
Pharmaceuticals, 2014 Histone deacetylases (HDACs) enzymes, which affect the acetylation status of histones and other important cellular proteins, have been recognized as potentially useful therapeutic targets for a broad range of human disorders.
Elizabeth A. Thomas
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Neurodegenerative models in Drosophila: Polyglutamine disorders, Parkinson disease, and amyotrophic lateral sclerosis [PDF]
Neurobiology of Disease, 2010 Neurodegenerative diseases encompass a large group of neurological disorders. Clinical symptoms can include memory loss, cognitive impairment, loss of movement or loss of control of movement, and loss of sensation.
Surendra S. Ambegaokar +2 more
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RNA toxicity in polyglutamine disorders: concepts, models, and progress of research [PDF]
Journal of Molecular Medicine, 2013 In Huntington's disease and other polyglutamine (polyQ) disorders, mutant proteins containing a long polyQ stretch are well documented as the trigger of numerous aberrant cellular processes that primarily lead to degeneration and, ultimately, the death of neuronal cells.
Agnieszka Fiszer +2 more
exaly +5 more sources
Unbiased human genomic characterization of polyglutamine disorder genes to guide biological understanding and therapeutic strategies [PDF]
HGG AdvancesSummary: Polyglutamine (polyQ) disorders, such as Huntington disease (HD) and several spinocerebellar ataxias, are severe neurological disorders caused by glutamine codon repeat expansions.
Kevin Lucy Namuli +2 more
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CAG repeat RNA as an auxiliary toxic agent in polyglutamine disorders. [PDF]
RNA Biol, 2011 Over 20 genetic loci with abnormal expansions of short tandem repeats have been associated with human hereditary neurological diseases. Of these, specific trinucleotide repeats located in non-coding and coding regions of individual genes implicated in these disorders are strongly overrepresented.
Wojciechowska M, Krzyzosiak WJ.
europepmc +4 more sources
Calpain-mediated proteolysis as driver and modulator of polyglutamine toxicity
Frontiers in Molecular Neuroscience, 2022 Among posttranslational modifications, directed proteolytic processes have the strongest impact on protein integrity. They are executed by a variety of cellular machineries and lead to a wide range of molecular consequences.
Rana Dilara Incebacak Eltemur +4 more
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Coiled-Coil Motifs of RNA-Binding Proteins: Dynamicity in RNA Regulation
Frontiers in Cell and Developmental Biology, 2020 Neuronal granules are biomolecular condensates that concentrate high quantities of RNAs and RNA-related proteins within neurons. These dense packets of information are trafficked from the soma to distal sites rich in polysomes, where local protein ...
Lenzie K. Ford, Luana Fioriti
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Pathogenesis of polyglutamine disorders: aggregation revisited [PDF]
Human Molecular Genetics, 2003 Expansion of CAG trinucleotide repeats coding for polyglutamine in unrelated proteins causes at least nine late-onset progressive neurodegenerative disorders, including Huntington's disease and a number of spinocerebellar ataxias. Expanded polyglutamine provokes a dominant gain-of-function neurotoxicity, regardless of the specific protein context ...
Michalik, A., Van Broeckhoven, C.
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eLife, 2020 Spinocerebellar ataxia type 3 (SCA3) belongs to the family of polyglutamine neurodegenerations. Each disorder stems from the abnormal lengthening of a glutamine repeat in a different protein. Although caused by a similar mutation, polyglutamine disorders
Sean L Johnson +4 more
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