ArticleHuman molecular genetics2016
Genomic removal of a therapeutic mini-dystrophin gene from adult mice elicits a Duchenne muscular dystrophy-like phenotype.
Article in Human molecular genetics, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed, 16 citations in OpenAlex.
- Modeling Reduced Contractility and Stiffness Using iPSC-Derived Cardiomyocytes Generated From Female Becker Muscular Dystrophy Carrier.JACC. Basic to translational science · 2023Article
- Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials.Human gene therapy · 2023Article
- Lifelong Outcomes of Systemic Adeno-Associated Virus Micro-Dystrophin Gene Therapy in a Murine Duchenne Muscular Dystrophy Model.Human gene therapy · 2023Article
- Large-scale sequencing of flatfish genomes provides insights into the polyphyletic origin of their specialized body plan.Nature genetics · 2021Article
- Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Dystrophin and calcium current are decreased in cardiomyocytes expressing Cre enzyme driven by αMHC but not TNT promoter.Scientific reports · 2019Article
- AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Questions Answered and Unanswered by the First CRISPR Editing Study in a Canine Model of Duchenne Muscular Dystrophy.Human gene therapy · 2019Article
- CRISPR alleviates muscular dystrophy in dogs.Nature biomedical engineering · 2018Article
- Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Review
- Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model.Human gene therapy · 2018Article
- Uniform low-level dystrophin expression in the heart partially preserved cardiac function in an aged mouse model of Duchenne cardiomyopathy.Journal of molecular and cellular cardiology · 2017Article
- Adeno-associated virus-mediated gene therapy for rare pediatric neurogenetic diseases: Current status and outlook.Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciencesArticle
Corrections and comments
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Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
Duchenne muscular dystrophy (DMD) is caused by dystrophin deficiency. A fundamental question in DMD pathogenesis and dystrophin gene therapy is whether muscle health depends on continuous dystrophin expression throughout the life. Published data suggest that transient dystrophin expression in early life might offer permanent protection. To study the consequences of adulthood dystrophin loss, we generated two strains of floxed mini-dystrophin transgenic mice on the dystrophin-null background. Muscle diseases were prevented in skeletal muscle of the YL238 strain and the heart of the SJ13 strain by selective expression of a therapeutic mini-dystrophin gene in skeletal muscle and heart, respectively. The mini-dystrophin gene was removed from the tibialis anterior (TA) muscle of 8-month-old YL238 mice and the heart of 7-month-old SJ13 mice using an adeno-associated virus serotype-9 Cre recombinase vector (AAV.CBA.Cre). At 12 and 15 months after AAV.CBA.Cre injection, mini-dystrophin expression was reduced by ∼87% in the TA muscle of YL238 mice and ∼64% in the heart of SJ13 mice. Mini-dystrophin reduction caused muscle atrophy, degeneration and force loss in the TA muscle of YL238 mice and significantly compromised left ventricular hemodynamics in SJ13 mice. Our results suggest that persistent dystrophin expression is essential for continuous muscle and heart protection.
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