ArticleProceedings of the National Academy of Sciences of the United States of America2002
Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 papers.
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Who cites it
46 citing papers in PubMed, 141 citations in OpenAlex.
- Genetic strategies for therapy of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids · 2025Review
- The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy.Molecular therapy. Methods & clinical development · 2022Article
- Dystrophin Cardiomyopathies: Clinical Management, Molecular Pathogenesis and Evolution towards Precision Medicine.Journal of clinical medicine · 2018Review
- Nanotherapy for Duchenne muscular dystrophy.Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology · 2018Review
- Superparamagnetic Iron Oxide Nanoparticles in Musculoskeletal Biology.Tissue engineering. Part B, Reviews · 2017Article
- Progress toward Gene Therapy for Duchenne Muscular Dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Review
- Gene transfer to the outflow tract.Experimental eye research · 2017Review
- Dystrophin Gene Replacement and Gene Repair Therapy for Duchenne Muscular Dystrophy in 2016: An Interview.Human gene therapy. Clinical development · 2016Article
- Genome Editing Gene Therapy for Duchenne Muscular Dystrophy.Journal of neuromuscular diseases · 2015Article
- How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse.Human molecular genetics · 2015Article
- Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy.Molecules (Basel, Switzerland) · 2015Review
- Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy.Disease models & mechanisms · 2015Review
- The FVB Background Does Not Dramatically Alter the Dystrophic Phenotype of Mdx Mice.PLoS currents · 2015Article
- Contemporary Animal Models For Human Gene Therapy Applications.Current gene therapy · 2015Review
- Biologic treatment of mild and moderate intervertebral disc degeneration.Molecular medicine (Cambridge, Mass.) · 2014Review
- Gene and cell-mediated therapies for muscular dystrophy.Muscle & nerve · 2013Review
- Capsid-modified adenoviral vectors for improved muscle-directed gene therapy.Human gene therapy · 2012Article
- Soluble miniagrin enhances contractile function of engineered skeletal muscle.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2012Article
- Effect of rapamycin on immunity induced by vector-mediated dystrophin expression in mdx skeletal muscle.Scientific reports · 2012Article
Corrections and comments
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Duchenne muscular dystrophy is a lethal X-linked recessive disorder caused by mutations in the dystrophin gene. Delivery of functionally effective levels of dystrophin to immunocompetent, adult mdx (dystrophin-deficient) mice has been challenging because of the size of the gene, immune responses against viral vectors, and inefficient infection of mature muscle. Here we show that high titer stocks of three different gutted adenoviral vectors carrying full-length, muscle-specific, dystrophin expression cassettes are able to efficiently transduce muscles of 1-yr-old mdx mice. Single i.m. injections of viral vector restored dystrophin production to 25-30% of mouse limb muscle 1 mo after injection. Furthermore, functional tests of virally transduced muscles revealed almost 40% correction of their high susceptibility to contraction-induced injury. Our results show that functional abnormalities of dystrophic muscle can be corrected by delivery of full-length dystrophin to adult, immunocompetent mdx mice, raising the prospects for gene therapy of muscular dystrophies.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.