Pigs with δ-sarcoglycan deficiency exhibit traits of genetic cardiomyopathy.

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ID: 100646
2020
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Abstract
Genetic cardiomyopathy is a group of intractable cardiovascular disorders involving heterogeneous genetic contribution. This heterogeneity has hindered the development of life-saving therapies for this serious disease. Genetic mutations in dystrophin and its associated glycoproteins cause cardiomuscular dysfunction. Large animal models incorporating these genetic defects are crucial for developing effective medical treatments, such as tissue regeneration and gene therapy. In the present study, we knocked out the δ-sarcoglycan (δ-SG) gene (SGCD) in domestic pig by using a combination of efficient de novo gene editing and somatic cell nuclear transfer. Loss of δ-SG expression in the SGCD knockout pigs caused a concomitant reduction in the levels of α-, β-, and γ-SG in the cardiac and skeletal sarcolemma, resulting in systolic dysfunction, myocardial tissue degeneration, and sudden death. These animals exhibited symptoms resembling human genetic cardiomyopathy and are thus promising for use in preclinical studies of next-generation therapies.
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matsunari2020pigslaboratory Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Matsunari, Hitomi;Honda, Michiyo;Watanabe, Masahito;Fukushima, Satsuki;Suzuki, Kouta;Miyagawa, Shigeru;Nakano, Kazuaki;Umeyama, Kazuhiro;Uchikura, Ayuko;Okamoto, Kazutoshi;Nagaya, Masaki;Toyo-Oka, Teruhiko;Sawa, Yoshiki;Nagashima, Hiroshi;
Journal laboratory investigation; a journal of technical methods and pathology
Year 2020
DOI
10.1038/s41374-020-0406-7
URL
Keywords
oxidative stress gene therapy dilated cardiomyopathy muscular dystrophy calcium heart phospholamban serca2a mdx membrane stabilization

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