ArticleResearch and reports in biology2011
Duchenne muscular dystrophy gene therapy: Lost in translation?
Article in Research and reports in biology, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
40 citing papers in PubMed, 55 citations in OpenAlex.
- Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy.Science translational medicine · 2012Trial
- Circulatory CCL2 distinguishes Duchenne muscular dystrophy dogs.Disease models & mechanisms · 2025Article
- Generation of a Dystrophin Mutant in Dog by Nuclear Transfer Using CRISPR/Cas9-Mediated Somatic Cells: A Preliminary Study.International journal of molecular sciences · 2022Article
- Dystrophin deficiency impairs vascular structure and function in the canine model of Duchenne muscular dystrophy.The Journal of pathology · 2021Article
- Widespread severe myodegeneration in a compound heterozygote female dog with dystrophin deficiency.Veterinary medicine and science · 2021Article
- Epigenetic modifications in muscle regeneration and progression of Duchenne muscular dystrophy.Clinical epigenetics · 2021Review
- Current Pharmacological Strategies for Duchenne Muscular Dystrophy.Frontiers in cell and developmental biology · 2021Review
- X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation.Skeletal muscle · 2020Article
- Gene Therapy for Heart Failure: New Perspectives.Current heart failure reports · 2018Review
- Nitric oxide-dependent attenuation of noradrenaline-induced vasoconstriction is impaired in the canine model of Duchenne muscular dystrophy.The Journal of physiology · 2018Article
- How will the field of gene therapy survive its success?Bioengineering & translational medicine · 2018Review
- Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model.Human gene therapy · 2018Article
- Non-invasive evaluation of muscle disease in the canine model of Duchenne muscular dystrophy by electrical impedance myography.PloS one · 2017Article
- Genome-wide association study to identify potential genetic modifiers in a canine model for Duchenne muscular dystrophy.BMC genomics · 2016Article
- Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method.Molecular therapy. Nucleic acids · 2016Article
- Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.Human molecular genetics · 2015Article
- Intra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune tolerance.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
- Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy.Disease models & mechanisms · 2015Review
- Duchenne muscular dystrophy gene therapy in the canine model.Human gene therapy. Clinical development · 2015Review
- The FVB Background Does Not Dramatically Alter the Dystrophic Phenotype of Mdx Mice.PLoS currents · 2015Article
Corrections and comments
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Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
A milestone of molecular medicine is the identification of dystrophin gene mutation as the cause of Duchenne muscular dystrophy (DMD). Over the last 2 decades, major advances in dystrophin biology and gene delivery technology have created an opportunity to treat DMD with gene therapy. Remarkable success has been achieved in treating dystrophic mice. Several gene therapy strategies, including plasmid transfer, exon skipping, and adeno-associated virus-mediated microdystrophin therapy, have entered clinical trials. However, therapeutic benefit has not been realized in DMD patients. Bridging the gap between mice and humans is no doubt the most pressing issue facing DMD gene therapy now. In contrast to mice, dystrophin-deficient dogs are genetically and phenotypically similar to human patients. Preliminary gene therapy studies in the canine model may offer critical insights that cannot be obtained from murine studies. It is clear that the canine DMD model may represent an important link between mice and humans. Unfortunately, our current knowledge of dystrophic dogs is limited, and the full picture of disease progression remains to be clearly defined. We also lack rigorous outcome measures (such as in situ force measurement) to monitor therapeutic efficacy in dystrophic dogs. Undoubtedly, maintaining a dystrophic dog colony is technically demanding, and the cost of dog studies cannot be underestimated. A carefully coordinated effort from the entire DMD community is needed to make the best use of the precious dog resource. Successful DMD gene therapy may depend on valid translational studies in dystrophin-deficient dogs.
Identifiers
What OpenQuestion holds
Registered trials
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.