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@ARTICLE{Bauer:143502,
      author       = {R. Bauer and H. Enns and A. Jungmann and B. Leuchs$^*$ and
                      C. Volz and S. Schinkel and W. J. Koch and P. W. Raake and
                      P. Most and H. A. Katus and O. J. Müller},
      title        = {{V}arious effects of {AAV}9-mediated β{ARK}ct gene therapy
                      on the heart in dystrophin-deficient (mdx) mice and
                      δ-sarcoglycan-deficient ({S}gcd-/-) mice.},
      journal      = {Neuromuscular disorders},
      volume       = {29},
      number       = {3},
      issn         = {0960-8966},
      address      = {Amsterdam [u.a.]},
      publisher    = {Elsevier Science},
      reportid     = {DKFZ-2019-01086},
      pages        = {231 - 241},
      year         = {2019},
      abstract     = {So far effective strategies to treat cardiomyopathy in
                      patients with muscular dystrophies are still not clearly
                      defined. Previously, treatment with β-blockers showed
                      beneficial effects on the development of cardiomyopathy in
                      dystrophin-deficient (mdx) mice, but not in
                      δ-sarcoglycan-deficient (Sgcd-/-) mice. We therefore aimed
                      to study a more specific approach to target maladaptive
                      β-adrenergic signalling in these mice. It has been shown
                      that lowering cardiac G-protein-coupled-receptor-kinase-2
                      (GRK2) activity with βARKct expression, a peptide inhibitor
                      of protein-coupled-receptor-kinase-2 (GRK2), results in
                      improvement of heart failure in several different animal
                      models. We therefore investigated whether adeno-associated
                      virus type 9 (AAV9)-mediated gene delivery of βARKct, could
                      ameliorate cardiac pathology in mdx and Sgcd-/- mice. We
                      found that long-term treatment with AAV9- βARKct-cDNA with
                      a cardiac-specific promoter significantly improves left
                      ventricular systolic function and reduces myocardial
                      hypertrophy in mdx mice, whereas only mild beneficial
                      effects on cardiac function is observed in Sgcd-/- mice.
                      Interestingly, in contrast to mdx mice neither GRK2 nor
                      nuclear-factor-kappaB (NFκB) were upregulated in Sgcd-/-
                      mice. Taken together, effectiveness of AAV-mediated βARKct
                      therapy may vary between different genetic mutations and
                      presumably depend on the state of adrenergic dysregulation
                      mediated through the upregulation of GRK2.},
      cin          = {F010},
      ddc          = {610},
      cid          = {I:(DE-He78)F010-20160331},
      pnm          = {316 - Infections and cancer (POF3-316)},
      pid          = {G:(DE-HGF)POF3-316},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:30782477},
      doi          = {10.1016/j.nmd.2018.12.006},
      url          = {https://inrepo02.dkfz.de/record/143502},
}