Various effects of AAV9-mediated βARKct gene therapy on the heart in dystrophin-deficient (mdx) mice and δ-sarcoglycan-deficient (Sgcd-/-) mice
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-/...
Gespeichert in:
| Hauptverfasser: | , , , , , , , , |
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| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
2019
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| In: |
Neuromuscular disorders
Year: 2019, Jahrgang: 29, Heft: 3, Pages: 231-241 |
| ISSN: | 1873-2364 |
| DOI: | 10.1016/j.nmd.2018.12.006 |
| Online-Zugang: | Verlag, Volltext: http://dx.doi.org/10.1016/j.nmd.2018.12.006
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| Verfasserangaben: | Ralf Bauer, Helene Enns, Andreas Jungmann, Barbara Leuchs, Christian Volz, Stefanie Schinkel, Walter J. Koch, Philip W. Raake, Patrick Most, Hugo A. Katus, Oliver J. Müller |
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| 245 | 1 | 0 | |a Various effects of AAV9-mediated βARKct gene therapy on the heart in dystrophin-deficient (mdx) mice and δ-sarcoglycan-deficient (Sgcd-/-) mice |c Ralf Bauer, Helene Enns, Andreas Jungmann, Barbara Leuchs, Christian Volz, Stefanie Schinkel, Walter J. Koch, Philip W. Raake, Patrick Most, Hugo A. Katus, Oliver J. Müller |
| 246 | 3 | 3 | |a Various effects of AAV9-mediated betaARKct gene therapy on the heart in dystrophin-deficient (mdx) mice and delta-sarcoglycan-deficient (Sgcd-/-) mice |
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| 520 | |a 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. | ||
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