Improvement of cardiac fibrosis in dystrophic mice by rAAV9-mediated microdystrophin transduction
JH Shin, Y Nitahara-Kasahara, H Hayashita-Kinoh… - Gene therapy, 2011 - nature.com
Duchenne muscular dystrophy (DMD) is the most common form of the progressive muscular
dystrophies characterized by defects of the dystrophin gene. Although primarily …
dystrophies characterized by defects of the dystrophin gene. Although primarily …
[HTML][HTML] AAV-microdystrophin therapy improves cardiac performance in aged female mdx mice
Dystrophin deficiency leads to lethal dilated Duchenne cardiomyopathy. A promising
therapy is to deliver a highly abbreviated microdystrophin gene to the heart using adeno …
therapy is to deliver a highly abbreviated microdystrophin gene to the heart using adeno …
Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice
Abstract Adeno-associated virus (AAV)-mediated microdystrophin gene therapy holds great
promise for treating Duchenne muscular dystrophy (DMD). Previous studies have revealed …
promise for treating Duchenne muscular dystrophy (DMD). Previous studies have revealed …
AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in> 21-m-old mdx mice, an end-stage model of Duchenne …
Duchenne muscular dystrophy (DMD) is a fatal genetic disease caused by the absence of
the sarcolemmal protein dystrophin. Dilated cardiomyopathy leading to heart failure is a …
the sarcolemmal protein dystrophin. Dilated cardiomyopathy leading to heart failure is a …
Microdystrophin Gene Therapy of Cardiomyopathy Restores Dystrophin-Glycoprotein Complex and Improves Sarcolemma Integrity in the Mdx Mouse Heart
Y Yue, Z Li, SQ Harper, RL Davisson, JS Chamberlain… - Circulation, 2003 - Am Heart Assoc
Background—More than 90% of Duchenne muscular dystrophy (DMD) patients develop
cardiomyopathy, and many die of cardiac failure. Despite tremendous progress in skeletal …
cardiomyopathy, and many die of cardiac failure. Despite tremendous progress in skeletal …
Long-Term Preservation of Cardiac Structure and Function After Adeno-Associated Virus Serotype 9-Mediated Microdystrophin Gene Transfer in mdx Mice
S Schinkel, R Bauer, R Bekeredjian, R Stucka… - Human gene …, 2012 - liebertpub.com
Dystrophin plays an important role in muscle contraction, linking the intracellular
cytoskeleton to the extracellular matrix. Mutations of the dystrophin gene leading to a …
cytoskeleton to the extracellular matrix. Mutations of the dystrophin gene leading to a …
[HTML][HTML] Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure
DW Townsend, MJ Blankinship, JM Allen… - Molecular Therapy, 2007 - cell.com
Duchenne muscular dystrophy (DMD) is a fatal disease of striated muscle deterioration
resulting from the loss of the cytoskeletal protein dystrophin. Most patients develop …
resulting from the loss of the cytoskeletal protein dystrophin. Most patients develop …
[HTML][HTML] Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model
AB Piepho, J Lowe, LR Cumby, LE Dorn… - … Therapy Methods & …, 2023 - cell.com
Micro-dystrophin gene replacement therapies for Duchenne muscular dystrophy (DMD) are
currently in clinical trials, but have not been thoroughly investigated for their efficacy on …
currently in clinical trials, but have not been thoroughly investigated for their efficacy on …
[HTML][HTML] SERCA2a gene transfer improves electrocardiographic performance in aged mdx mice
Background Cardiomyocyte calcium overloading has been implicated in the pathogenesis of
Duchenne muscular dystrophy (DMD) heart disease. The cardiac isoform of sarcoplasmic …
Duchenne muscular dystrophy (DMD) heart disease. The cardiac isoform of sarcoplasmic …
Gene therapy rescues cardiac dysfunction in Duchenne muscular dystrophy mice by elevating cardiomyocyte deoxy-adenosine triphosphate
SC Kolwicz, JK Hall, F Moussavi-Harami… - JACC: Basic to …, 2019 - jacc.org
Mutations in the gene encoding for dystrophin leads to structural and functional deterioration
of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy …
of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy …