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@ARTICLE{Matthes:857532,
      author       = {Matthes, Frank and Massari, Serena and Bochicchio, Anna and
                      Schorpp, Kenji and Schilling, Judith and Weber, Stephanie
                      and Offermann, Nina and Desantis, Jenny and Wanker, Erich
                      and Carloni, Paolo and Hadian, Kamyar and Tabarrini, Oriana
                      and Rossetti, Giulia and Krauss, Sybille},
      title        = {{R}educing {M}utant {H}untingtin {P}rotein {E}xpression in
                      {L}iving {C}ells by a {N}ewly {I}dentified {RNA} {CAG}
                      {B}inder},
      journal      = {ACS chemical neuroscience},
      volume       = {9},
      number       = {6},
      issn         = {1948-7193},
      address      = {Washington, DC},
      publisher    = {ACS Publ.},
      reportid     = {FZJ-2018-06523},
      pages        = {1399 - 1408},
      year         = {2018},
      abstract     = {Expanded CAG trinucleotide repeats in Huntington’s
                      disease (HD) are causative for neurotoxicity. The mutant CAG
                      repeat RNA encodes neurotoxic polyglutamine proteins and can
                      lead to a toxic gain of function by aberrantly recruiting
                      RNA-binding proteins. One of these is the MID1 protein,
                      which induces aberrant Huntingtin (HTT) protein translation
                      upon binding. Here we have identified a set of CAG repeat
                      binder candidates by in silico methods. One of those,
                      furamidine, reduces the level of binding of HTT mRNA to MID1
                      and other target proteins in vitro. Metadynamics
                      calculations, fairly consistent with experimental data
                      measured here, provide hints about the binding mode of the
                      ligand. Importantly, furamidine also decreases the protein
                      level of HTT in a HD cell line model. This shows that small
                      molecules masking RNA–MID1 interactions may be active
                      against mutant HTT protein in living cells.},
      cin          = {IAS-5 / JARA-HPC / JSC / INM-9},
      ddc          = {540},
      cid          = {I:(DE-Juel1)IAS-5-20120330 / $I:(DE-82)080012_20140620$ /
                      I:(DE-Juel1)JSC-20090406 / I:(DE-Juel1)INM-9-20140121},
      pnm          = {574 - Theory, modelling and simulation (POF3-574) / 511 -
                      Computational Science and Mathematical Methods (POF3-511)},
      pid          = {G:(DE-HGF)POF3-574 / G:(DE-HGF)POF3-511},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:29506378},
      UT           = {WOS:000436211800022},
      doi          = {10.1021/acschemneuro.8b00027},
      url          = {https://juser.fz-juelich.de/record/857532},
}