ArticleSkeletal muscle2015
Mesoangioblast delivery of miniagrin ameliorates murine model of merosin-deficient congenital muscular dystrophy type 1A.
Article in Skeletal muscle, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 17 citations in OpenAlex.
- Progress on cell therapy for skeletal muscle disorders.Advanced drug delivery reviews · 2026Review
- Extracellular matrix in skeletal muscle injury and atrophy: mechanisms and therapeutic implications.Journal of orthopaedic translation · 2025Review
- Merosin deficient congenital muscular dystrophy type 1A: An international workshop on the road to therapy 15-17 November 2019, Maastricht, the Netherlands.Neuromuscular disorders : NMD · 2021Article
- Myogenic Cell Transplantation in Genetic and Acquired Diseases of Skeletal Muscle.Frontiers in genetics · 2021Review
- LAMA2 Neuropathies: Human Findings and Pathomechanisms From Mouse Models.Frontiers in molecular neuroscience · 2020Article
- A Family of Laminin α2 Chain-Deficient Mouse Mutants: Advancing the Research on LAMA2-CMD.Frontiers in molecular neuroscience · 2020Review
- Pericytes in Muscular Dystrophies.Advances in experimental medicine and biology · 2019Article
- Current understanding and treatment of cardiac and skeletal muscle pathology in laminin-α2 chain-deficient congenital muscular dystrophy.The application of clinical genetics · 2019Article
- At the Crossroads of Clinical and Preclinical Research for Muscular Dystrophy-Are We Closer to Effective Treatment for Patients?International journal of molecular sciences · 2018Review
- Preclinical and clinical advances in transposon-based gene therapy.Bioscience reports · 2017Review
- piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.Nucleic acids research · 2016Article
- Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy.EMBO molecular medicine · 2015Article
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Authors and funding
12 authors at 6 institutions in 3 countries.
Funding
Abstract
backgroundMerosin-deficient congenital muscular dystrophy type-1A (MDC1A) is characterized by progressive muscular dystrophy and dysmyelinating neuropathy caused by mutations of the α2 chain of laminin-211, the predominant laminin isoform of muscles and nerves. MDC1A has no available treatment so far, although preclinical studies showed amelioration of the disease by the overexpression of miniagrin (MAG). MAG reconnects orphan laminin-211 receptors to other laminin isoforms available in the extracellular matrix of MDC1A mice.
methodsMesoangioblasts (MABs) are vessel-associated progenitors that can form the skeletal muscle and have been shown to restore defective protein levels and motor skills in animal models of muscular dystrophies. As gene therapy in humans still presents challenging technical issues and limitations, we engineered MABs to overexpress MAG to treat MDC1A mouse models, thus combining cell to gene therapy.
resultsMABs synthesize and secrete only negligible amount of laminin-211 either in vitro or in vivo. MABs engineered to deliver MAG and injected in muscles of MDC1A mice showed amelioration of muscle histology, increased expression of laminin receptors in muscle, and attenuated deterioration of motor performances. MABs did not enter the peripheral nerves, thus did not affect the associated peripheral neuropathy.
conclusionsOur study demonstrates the potential efficacy of combining cell with gene therapy to treat MDC1A.
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