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000143502 0247_ $$2doi$$a10.1016/j.nmd.2018.12.006
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000143502 1001_ $$aBauer, Ralf$$b0
000143502 245__ $$aVarious effects of AAV9-mediated βARKct gene therapy on the heart in dystrophin-deficient (mdx) mice and δ-sarcoglycan-deficient (Sgcd-/-) mice.
000143502 260__ $$aAmsterdam [u.a.]$$bElsevier Science$$c2019
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000143502 520__ $$aSo far effective strategies to treat cardiomyopathy in patients with muscular dystrophies are still not clearly defined. Previously, treatment with β-blockers showed beneficial effects on the development of cardiomyopathy in dystrophin-deficient (mdx) mice, but not in δ-sarcoglycan-deficient (Sgcd-/-) mice. We therefore aimed to study a more specific approach to target maladaptive β-adrenergic signalling in these mice. It has been shown that lowering cardiac G-protein-coupled-receptor-kinase-2 (GRK2) activity with βARKct expression, a peptide inhibitor of protein-coupled-receptor-kinase-2 (GRK2), results in improvement of heart failure in several different animal models. We therefore investigated whether adeno-associated virus type 9 (AAV9)-mediated gene delivery of βARKct, could ameliorate cardiac pathology in mdx and Sgcd-/- mice. We found that long-term treatment with AAV9- βARKct-cDNA with a cardiac-specific promoter significantly improves left ventricular systolic function and reduces myocardial hypertrophy in mdx mice, whereas only mild beneficial effects on cardiac function is observed in Sgcd-/- mice. Interestingly, in contrast to mdx mice neither GRK2 nor nuclear-factor-kappaB (NFκB) were upregulated in Sgcd-/- mice. Taken together, effectiveness of AAV-mediated βARKct therapy may vary between different genetic mutations and presumably depend on the state of adrenergic dysregulation mediated through the upregulation of GRK2.
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000143502 7001_ $$aEnns, Helene$$b1
000143502 7001_ $$aJungmann, Andreas$$b2
000143502 7001_ $$0P:(DE-He78)119d7d540a0e6fc5734898bef4866d7f$$aLeuchs, Barbara$$b3$$udkfz
000143502 7001_ $$aVolz, Christian$$b4
000143502 7001_ $$aSchinkel, Stefanie$$b5
000143502 7001_ $$aKoch, Walter J$$b6
000143502 7001_ $$aRaake, Philip W$$b7
000143502 7001_ $$aMost, Patrick$$b8
000143502 7001_ $$aKatus, Hugo A$$b9
000143502 7001_ $$aMüller, Oliver J$$b10
000143502 773__ $$0PERI:(DE-600)2008287-3$$a10.1016/j.nmd.2018.12.006$$gVol. 29, no. 3, p. 231 - 241$$n3$$p231 - 241$$tNeuromuscular disorders$$v29$$x0960-8966$$y2019
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