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@ARTICLE{Shahriyari:165318,
      author       = {Shahriyari, Mina and Islam, Rezaul and Sakib, Sadman M and
                      Rinn, Malte and Rika, Anastasia and Krüger, Dennis and
                      Kaurani, Lalit and Gisa, Verena and Winterhoff, Mandy and
                      Anandakumar, Harithaa and Shomroni, Orr and Schmidt,
                      Matthias and Salinas, Gabriela and Unger, Andreas and Linke,
                      Wolfgang A and Zschüntzsch, Jana and Schmidt, Jens and
                      Bassel-Duby, Rhonda and Olson, Eric N and Fischer, André
                      and Zimmermann, Wolfram-Hubertus and Tiburcy, Malte},
      title        = {{E}ngineered skeletal muscle recapitulates human muscle
                      development, regeneration and dystrophy.},
      journal      = {Journal of cachexia, sarcopenia and muscle},
      volume       = {13},
      number       = {6},
      issn         = {2190-5991},
      address      = {Hoboken, NJ},
      publisher    = {Wiley},
      reportid     = {DZNE-2022-01596},
      pages        = {3106-3121},
      year         = {2022},
      abstract     = {Human pluripotent stem cell-derived muscle models show
                      great potential for translational research. Here, we
                      describe developmentally inspired methods for the derivation
                      of skeletal muscle cells and their utility in skeletal
                      muscle tissue engineering with the aim to model skeletal
                      muscle regeneration and dystrophy in vitro.Key steps include
                      the directed differentiation of human pluripotent stem cells
                      to embryonic muscle progenitors followed by primary and
                      secondary foetal myogenesis into three-dimensional muscle.
                      To simulate Duchenne muscular dystrophy (DMD), a
                      patient-specific induced pluripotent stem cell line was
                      compared to a CRISPR/Cas9-edited isogenic control line.The
                      established skeletal muscle differentiation protocol
                      robustly and faithfully recapitulates critical steps of
                      embryonic myogenesis in two-dimensional and
                      three-dimensional cultures, resulting in functional human
                      skeletal muscle organoids (SMOs) and engineered skeletal
                      muscles (ESMs) with a regeneration-competent satellite-like
                      cell pool. Tissue-engineered muscle exhibits organotypic
                      maturation and function (up to 5.7 ± 0.5 mN tetanic twitch
                      tension at 100 Hz in ESM). Contractile performance could be
                      further enhanced by timed thyroid hormone treatment,
                      increasing the speed of contraction (time to peak
                      contraction) as well as relaxation (time to $50\%$
                      relaxation) of single twitches from 107 ± 2 to 75 ± 4 ms
                      (P < 0.05) and from 146 ± 6 to 100 ± 6 ms (P < 0.05),
                      respectively. Satellite-like cells could be documented as
                      largely quiescent PAX7+ cells (75 ± $6\%$ Ki67- ) located
                      adjacent to muscle fibres confined under a
                      laminin-containing basal membrane. Activation of the
                      engineered satellite-like cell niche was documented in a
                      cardiotoxin injury model with marked recovery of
                      contractility to 57 ± $8\%$ of the pre-injury force 21 days
                      post-injury (P < 0.05 compared to Day 2 post-injury), which
                      was completely blocked by preceding irradiation. Absence of
                      dystrophin in DMD ESM caused a marked reduction of
                      contractile force (-35 ± $7\%,$ P < 0.05) and impaired
                      expression of fast myosin isoforms resulting in prolonged
                      contraction (175 ± 14 ms, P < 0.05 vs. gene-edited control)
                      and relaxation (238 ± 22 ms, P < 0.05 vs. gene-edited
                      control) times. Restoration of dystrophin levels by gene
                      editing rescued the DMD phenotype in ESM.We introduce human
                      muscle models with canonical properties of bona fide
                      skeletal muscle in vivo to study muscle development,
                      maturation, disease and repair.},
      keywords     = {Humans / Muscular Dystrophy, Duchenne: genetics / Muscle,
                      Skeletal: metabolism / Muscle Development: genetics /
                      Satellite Cells, Skeletal Muscle: metabolism / Muscle
                      Fibers, Skeletal: metabolism / Duchenne muscular dystrophy
                      (Other) / hypaxial dermomyotome (Other) / limb muscle
                      (Other) / satellite cells (Other) / skeletal muscle organoid
                      (Other) / somite (Other) / tissue engineering (Other)},
      cin          = {AG Fischer 1 / Bioinformatics and Genome Dynamics Core},
      ddc          = {610},
      cid          = {I:(DE-2719)1410002 / I:(DE-2719)1440016},
      pnm          = {352 - Disease Mechanisms (POF4-352)},
      pid          = {G:(DE-HGF)POF4-352},
      typ          = {PUB:(DE-HGF)16},
      pmc          = {pmc:PMC9745484},
      pubmed       = {pmid:36254806},
      doi          = {10.1002/jcsm.13094},
      url          = {https://pub.dzne.de/record/165318},
}