Relevant aspects of golden retriever muscular dystrophy for the study of Duchenne muscular dystrophy in humans

被引:1
|
作者
Engracia de Moraes, Julieta Rodini [1 ]
Mouri Malvestio, Lygia Maria [2 ]
Martins, Isabela Mancini [1 ]
Erdmann Mosko, Patricia Regina [3 ]
Engracia Filho, Jair Rodini [3 ]
de Moraes, Flavio Ruas [1 ]
机构
[1] Univ Estadual Paulista UNESP, FCAV, BR-14884900 Jaboticabal, SP, Brazil
[2] Escola Med Vet, Fac Merid IMED, Passo Fundo, RS, Brazil
[3] Pontificia Univ Catolica Parana PUC PR, PPGCA, Curitiba, PR, Brazil
来源
CIENCIA RURAL | 2017年 / 47卷 / 10期
基金
巴西圣保罗研究基金会;
关键词
Duchenne muscular dystrophy; animal model; dystrophic Golden Retriever; ANIMAL-MODELS; MUSCLE FUNCTION; CANINE MODEL; STEM-CELLS; GENE; CARDIOMYOPATHY; EXPRESSION; TRANSDUCTION; INJECTION; MUTATION;
D O I
10.1590/0103-8478cr20160470
中图分类号
S3 [农学(农艺学)];
学科分类号
0901 ;
摘要
Golden Retriever muscular dystrophy (GRMD) is the most representative model for studying Duchenne muscular dystrophy (DMD) in humans, owing its phenotypic expression. DMD is a recessive disorder linked to the X chromosome in which the loss of dystrophin induces progressive weakness and degeneration of the skeletal and cardiac muscles, which lead to replacement by connective and adipose tissues. Onset of clinical signs occurs between 2 and 5 years of age, and many patients die from heart or respiratory failure. The main studies concerning dystrophic Golden Retrievers (DGR) sought to elucidate the pathophysiology of the disease and its clinical implications to develop therapies and alternative treatments to improve the quality of life and increase longevity of DMD patients. This review presents an overview of relevant contributions of the DGR model for elucidating DMD in humans.
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页数:11
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