ArticlePloS one2011
Worsening of cardiomyopathy using deflazacort in an animal model rescued by gene therapy.
Article in PloS one, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 19 citations in OpenAlex.
- The effect of atrial preference pacing on atrial fibrillation electrophysiological substrate in Myotonic Dystrophy type 1 population.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2014Trial
- Atrial fibrillation burden in Myotonic Dystrophy type 1 patients implanted with dual chamber pacemaker: the efficacy of the overdrive atrial algorithm at 2 year follow-up.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2013Trial
- Right atrial preference pacing algorithm in the prevention of paroxysmal atrial fibrillation in myotonic dystrophy type 1 patients: a long term follow-up study.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2012Trial
- Adeno-associated virus vector intraperitoneal injection induces colonic mucosa and submucosa transduction and alters the diversity and composition of the faecal microbiota in rats.Frontiers in cellular and infection microbiology · 2022Article
- Increased heterogeneity of ventricular repolarization in myotonic dystrophy type 1 population.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2016Article
- Adenosine-induced sinus tachycardia in a patient with Myotonic Dystrophy type 1.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2014Article
- Far field R-wave sensing in Myotonic Dystrophy type 1: right atrial appendage versus Bachmann's bundle region lead placement.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2014Article
- Treatment of dystrophin cardiomyopathies.Nature reviews. Cardiology · 2014Review
- Use of a lower dosage liver-detargeted AAV vector to prevent hamster muscular dystrophy.Human gene therapy · 2013Article
- Increased dispersion of ventricular repolarization in Emery Dreifuss muscular dystrophy patients.Medical science monitor : international medical journal of experimental and clinical research · 2012Article
- Improvement of survival in Duchenne Muscular Dystrophy: retrospective analysis of 835 patients.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2012Article
- The heart and cardiac pacing in Steinert disease.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2012Review
- Improving the course of muscular dystrophy?Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2012Article
- Treatment of dystrophinopathic cardiomyopathy: review of the literature and personal results.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2012Review
- Glucocorticoid-treated mice are an inappropriate positive control for long-term preclinical studies in the mdx mouse.PloS one · 2012Article
Corrections and comments
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Authors and funding
13 authors at 5 institutions in 1 country.
Funding
Abstract
We have previously demonstrated that gene therapy can rescue the phenotype and extend lifespan in the delta-sarcoglycan deficient cardiomyopathic hamster. In patients with similar genetic defects, steroids have been largely used to slow down disease progression. Aim of our study was to evaluate the combined effects of steroid treatment and gene therapy on cardiac function. We injected the human delta-sarcoglycan cDNA by adeno-associated virus (AAV) 2/8 by a single intraperitoneal injection into BIO14.6 Syrian hamsters at ten days of age to rescue the phenotype. We then treated the hamsters with deflazacort. Treatment was administered to half of the hamsters that had received the AAV and the other hamsters without AAV, as well as to normal hamsters. Both horizontal and vertical activities were greatly enhanced by deflazacort in all groups. As in previous experiments, the AAV treatment alone was able to preserve the ejection fraction (70±7% EF). However, the EF value declined (52±14%) with a combination of AAV and deflazacort. This was similar with all the other groups of affected animals. We confirm that gene therapy improves cardiac function in the BIO14.6 hamsters. Our results suggest that deflazacort is ineffective and may also have a negative impact on the cardiomyopathy rescue, possibly by boosting motor activity. This is unexpected and may have significance in terms of the lifestyle recommendations for patients.
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