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Conformational targeting of fibrillar polyglutamine proteins in live cells escalates aggregation and cytotoxicity.

PloS one | 2009

Misfolding- and aggregation-prone proteins underlying Parkinson's, Huntington's and Machado-Joseph diseases, namely alpha-synuclein, huntingtin, and ataxin-3 respectively, adopt numerous intracellular conformations during pathogenesis, including globular intermediates and insoluble amyloid-like fibrils. Such conformational diversity has complicated research into amyloid-associated intracellular dysfunction and neurodegeneration. To this end, recombinant single-chain Fv antibodies (scFvs) are compelling molecular tools that can be selected against specific protein conformations, and expressed inside cells as intrabodies, for investigative and therapeutic purposes.

Pubmed ID: 19492089 RIS Download

Research resources used in this publication

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Associated grants

  • Agency: NINDS NIH HHS, United States
    Id: R21 NS061257
  • Agency: NINDS NIH HHS, United States
    Id: NS061257
  • Agency: NINDS NIH HHS, United States
    Id: NS053912
  • Agency: NINDS NIH HHS, United States
    Id: R01 NS053912-04
  • Agency: NINDS NIH HHS, United States
    Id: R01 NS053912

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SPIP (tool)

RRID:SCR_009410

Software application that simulate pedigrees and genetic data in age-structured populations (entry from Genetic Analysis Software)

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BD CellQuest Pro (tool)

RRID:SCR_014489

A software for acquiring and analyzing flow cytometry data. The user can create plots with elements like regions, gates, statistics, markers, and annotated text. The software can also be used for batch analysis or quality control of analysis. Results can be exported and saved.

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