[HTML][HTML] Single SERCA2a therapy ameliorated dilated cardiomyopathy for 18 months in a mouse model of Duchenne muscular dystrophy

NB Wasala, Y Yue, W Lostal, LP Wasala, N Niranjan… - Molecular therapy, 2020 - cell.com
Loss of dystrophin leads to Duchenne muscular dystrophy (DMD). A pathogenic feature of
DMD is the significant elevation of cytosolic calcium. Supraphysiological calcium triggers …

[HTML][HTML] SERCA2a gene transfer improves electrocardiographic performance in aged mdx mice

JH Shin, B Bostick, Y Yue, R Hajjar, D Duan - Journal of translational …, 2011 - Springer
Background Cardiomyocyte calcium overloading has been implicated in the pathogenesis of
Duchenne muscular dystrophy (DMD) heart disease. The cardiac isoform of sarcoplasmic …

[HTML][HTML] Mitigation of muscular dystrophy in mice by SERCA overexpression in skeletal muscle

SA Goonasekera, CK Lam, DP Millay… - The Journal of …, 2011 - Am Soc Clin Investig
Muscular dystrophies (MDs) comprise a group of degenerative muscle disorders
characterized by progressive muscle wasting and often premature death. The primary defect …

[HTML][HTML] Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatments

C Vitiello, S Faraso, NC Sorrentino, G Di Salvo… - PLoS …, 2009 - journals.plos.org
Background The BIO14. 6 hamster is an excellent animal model for inherited
cardiomyopathy, because of its lethal and well-documented course, due to a spontaneous …

Overexpression of SERCA1a in the mdx Diaphragm Reduces Susceptibility to Contraction-Induced Damage

KJ Morine, MM Sleeper, ER Barton… - Human gene …, 2010 - liebertpub.com
Although the precise pathophysiological mechanism of muscle damage in dystrophin-
deficient muscle remains disputed, calcium appears to be a critical mediator of the …

[HTML][HTML] TRPC3, but not TRPC1, as a good therapeutic target for standalone or complementary treatment of DMD

A Creisméas, C Gazaille, A Bourdon… - Journal of translational …, 2021 - Springer
Background Duchenne muscular dystrophy (DMD) is an X-linked inherited disease caused
by mutations in the gene encoding dystrophin that leads to a severe and ultimately life …

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 …

B Bostick, JH Shin, Y Yue, NB Wasala, Y Lai… - Journal of molecular and …, 2012 - Elsevier
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 …

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 …

SERCA1 overexpression minimizes skeletal muscle damage in dystrophic mouse models

DAG Mázala, SJP Pratt, D Chen… - … of Physiology-Cell …, 2015 - journals.physiology.org
Duchenne muscular dystrophy (DMD) is characterized by progressive muscle wasting
secondary to repeated muscle damage and inadequate repair. Elevations in intracellular …

Dwarf open reading frame (DWORF) gene therapy ameliorated duchenne muscular dystrophy cardiomyopathy in aged mdx mice

ED Morales, Y Yue, TB Watkins, J Han… - Journal of the …, 2023 - Am Heart Assoc
Background Cardiomyopathy is a leading health threat in Duchenne muscular dystrophy
(DMD). Cytosolic calcium upregulation is implicated in DMD cardiomyopathy. Calcium is …