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

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

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;
laboratory investigation; a journal of technical methods and pathology 2020
284
matsunari2020pigslaboratory

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.

Citation

ID: 100646
Ref Key: matsunari2020pigslaboratory
Use this key to autocite in SciMatic or Thesis Manager

References

Blockchain Verification

Account:
NFT Contract Address:
0x95644003c57E6F55A65596E3D9Eac6813e3566dA
Article ID:
100646
Unique Identifier:
10.1038/s41374-020-0406-7
Network:
Scimatic Chain (ID: 481)
Loading...
Blockchain Readiness Checklist
Authors
Abstract
Journal Name
Year
Title
5/5
Creates 1,000,000 NFT tokens for this article
Token Features:
  • ERC-1155 Standard NFT
  • 1 Million Supply per Article
  • Transferable via MetaMask
  • Permanent Blockchain Record
Blockchain QR Code
Scan with Saymatik Web3.0 Wallet

Saymatik Web3.0 Wallet