ArticleProceedings of the National Academy of Sciences of the United States of America2018
Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2018. 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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13 citing papers in PubMed, 19 citations in OpenAlex.
- Is dystrophin immunogenicity a barrier to advancing gene therapy for Duchenne muscular dystrophy?Gene therapy · 2025Review
- Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Review
- Translating musculoskeletal bioengineering into tissue regeneration therapies.Science translational medicine · 2022Review
- Time to diagnosis of Duchenne muscular dystrophy remains unchanged: Findings from the Muscular Dystrophy Surveillance, Tracking, and Research Network, 2000-2015.Muscle & nerve · 2022Article
- Nucleic acid functionalized extracellular vesicles as promising therapeutic systems for nanomedicine.Extracellular vesicles and circulating nucleic acids · 2022Review
- The new challenge of "exercise + X″ therapy for Duchenne muscular dystrophy-Individualized identification of exercise tolerance and precise implementation of exercise intervention.Frontiers in physiology · 2022Review
- Improved transduction of canine X-linked muscular dystrophy with rAAV9-microdystrophin via multipotent MSC pretreatment.Molecular therapy. Methods & clinical development · 2021Article
- Article
- Engineered skeletal muscles for disease modeling and drug discovery.Biomaterials · 2019Review
- A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies-In Vitro and In Vivo Treatments to Boost Cellular Engraftment.International journal of molecular sciences · 2019Review
- Novel Therapies for Prevention and Early Treatment of Cardiomyopathies.Circulation research · 2019Review
- Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches.Journal of personalized medicine · 2019Review
- Building immune tolerance through DNA vaccination.Proceedings of the National Academy of Sciences of the United States of America · 2018Article
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12 authors at 5 institutions in 1 country.
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Abstract
In gene therapy for Duchenne muscular dystrophy there are two potential immunological obstacles. An individual with Duchenne muscular dystrophy has a genetic mutation in dystrophin, and therefore the wild-type protein is "foreign," and thus potentially immunogenic. The adeno-associated virus serotype-6 (AAV6) vector for delivery of dystrophin is a viral-derived vector with its own inherent immunogenicity. We have developed a technology where an engineered plasmid DNA is delivered to reduce autoimmunity. We have taken this approach into humans, tolerizing to myelin proteins in multiple sclerosis and to proinsulin in type 1 diabetes. Here, we extend this technology to a model of gene therapy to reduce the immunogenicity of the AAV vector and of the wild-type protein product that is missing in the genetic disease. Following gene therapy with systemic administration of recombinant AAV6-microdystrophin to mdx/mTR
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