% IMPORTANT: The following is UTF-8 encoded.  This means that in the presence
% of non-ASCII characters, it will not work with BibTeX 0.99 or older.
% Instead, you should use an up-to-date BibTeX implementation like “bibtex8” or
% “biber”.

@ARTICLE{Weykopf:140517,
      author       = {Weykopf, Beatrice and Haupt, Simone and Jungverdorben,
                      Johannes and Flitsch, Lea Jessica and Hebisch, Matthias and
                      Liu, Guang-Hui and Suzuki, Keiichiro and Belmonte, Juan
                      Carlos Izpisua and Peitz, Michael and Blaess, Sandra and
                      Till, Andreas and Brüstle, Oliver},
      title        = {{I}nduced pluripotent stem cell-based modeling of mutant
                      {LRRK}2-associated {P}arkinson's disease.},
      journal      = {European journal of neuroscience},
      volume       = {49},
      number       = {4},
      issn         = {0953-816X},
      address      = {Oxford [u.a.]},
      publisher    = {Blackwell},
      reportid     = {DZNE-2020-06839},
      pages        = {561-589},
      year         = {2019},
      abstract     = {Recent advances in cell reprogramming have enabled
                      assessment of disease-related cellular traits in
                      patient-derived somatic cells, thus providing a versatile
                      platform for disease modeling and drug development. Given
                      the limited access to vital human brain cells, this
                      technology is especially relevant for neurodegenerative
                      disorders such as Parkinson's disease (PD) as a tool to
                      decipher underlying pathomechanisms. Importantly, recent
                      progress in genome-editing technologies has provided an
                      ability to analyze isogenic induced pluripotent stem cell
                      (iPSC) pairs that differ only in a single genetic change,
                      thus allowing a thorough assessment of the molecular and
                      cellular phenotypes that result from monogenetic risk
                      factors. In this review, we summarize the current state of
                      iPSC-based modeling of PD with a focus on leucine-rich
                      repeat kinase 2 (LRRK2), one of the most prominent
                      monogenetic risk factors for PD linked to both familial and
                      idiopathic forms. The LRRK2 protein is a primarily cytosolic
                      multi-domain protein contributing to regulation of several
                      pathways including autophagy, mitochondrial function,
                      vesicle transport, nuclear architecture and cell morphology.
                      We summarize iPSC-based studies that contributed to
                      improving our understanding of the function of LRRK2 and its
                      variants in the context of PD etiopathology. These data,
                      along with results obtained in our own studies, underscore
                      the multifaceted role of LRRK2 in regulating cellular
                      homeostasis on several levels, including proteostasis,
                      mitochondrial dynamics and regulation of the cytoskeleton.
                      Finally, we expound advantages and limitations of
                      reprogramming technologies for disease modeling and drug
                      development and provide an outlook on future challenges and
                      expectations offered by this exciting technology.},
      subtyp        = {Review Article},
      keywords     = {Humans / Induced Pluripotent Stem Cells / Leucine-Rich
                      Repeat Serine-Threonine Protein Kinase-2: genetics /
                      Mitophagy / Models, Neurological / Parkinson Disease:
                      genetics / Parkinson Disease: therapy},
      cin          = {Cell Programming Unit},
      ddc          = {610},
      cid          = {I:(DE-2719)1013013},
      pnm          = {344 - Clinical and Health Care Research (POF3-344)},
      pid          = {G:(DE-HGF)POF3-344},
      typ          = {PUB:(DE-HGF)16},
      pmc          = {pmc:PMC7114274},
      pubmed       = {pmid:30656775},
      doi          = {10.1111/ejn.14345},
      url          = {https://pub.dzne.de/record/140517},
}