ReviewExpert reviews in molecular medicine2009
Emerging strategies for cell and gene therapy of the muscular dystrophies.
Review in Expert reviews in molecular medicine, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 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
35 citing papers in PubMed, 73 citations in OpenAlex.
- Biofabrication of a 3D human skeletal muscle microenvironment to study the early steps of fibrosis.Materials today. Bio · 2025Article
- CRISPR/Cas9-TargetedJournal of developmental biology · 2025Article
- The effect of hypoxia on myogenic differentiation and multipotency of the skeletal muscle-derived stem cells in mice.Stem cell research & therapy · 2022Article
- Computational Models Provide Insight into In Vivo Studies and Reveal the Complex Role of Fibrosis in mdx Muscle Regeneration.Annals of biomedical engineering · 2021Article
- Quantifying the economic impact of caregiving for Duchenne muscular dystrophy (DMD) in Spain.The European journal of health economics : HEPAC : health economics in prevention and care · 2020Article
- Developmentally regulated alternative splicing is perturbed in type 1 diabetic skeletal muscle.Muscle & nerve · 2017Article
- Prosurvival Factors Improve Functional Engraftment of Myogenically Converted Dermal Cells into Dystrophic Skeletal Muscle.Stem cells and development · 2016Article
- Induced Pluripotent Stem Cells derived Muscle Progenitors Effectively Mitigate Muscular Dystrophy through Restoring the Dystrophin Distribution.Journal of stem cell research & therapy · 2016Article
- Pharmacological therapeutics targeting the secondary defects and downstream pathology of Duchenne muscular dystrophy.Expert opinion on orphan drugs · 2016Article
- Multiscale models of skeletal muscle reveal the complex effects of muscular dystrophy on tissue mechanics and damage susceptibility.Interface focus · 2015Article
- Coaxing stem cells for skeletal muscle repair.Advanced drug delivery reviews · 2015Review
- Conserved regions of the DMD 3' UTR regulate translation and mRNA abundance in cultured myotubes.Neuromuscular disorders : NMD · 2014Article
- Engraftment potential of dermal fibroblasts following in vivo myogenic conversion in immunocompetent dystrophic skeletal muscle.Molecular therapy. Methods & clinical development · 2014Article
- Tranilast administration reduces fibrosis and improves fatigue resistance in muscles of mdx dystrophic mice.Fibrogenesis & tissue repair · 2014Article
- Novel and optimized strategies for inducing fibrosis in vivo: focus on Duchenne Muscular Dystrophy.Skeletal muscle · 2014Article
- Genetic manipulation of myoblasts and a novel primary myosatellite cell culture system: comparing and optimizing approaches.The FEBS journal · 2013Article
- Dystrophin's tandem calponin-homology domains: is the case closed?Biophysical journal · 2012Article
- The N-terminal actin-binding tandem calponin-homology (CH) domain of dystrophin is in a closed conformation in solution and when bound to F-actin.Biophysical journal · 2012Article
- Deletion of periostin reduces muscular dystrophy and fibrosis in mice by modulating the transforming growth factor-β pathway.Proceedings of the National Academy of Sciences of the United States of America · 2012Article
- Thermodynamic stability, unfolding kinetics, and aggregation of the N-terminal actin-binding domains of utrophin and dystrophin.Proteins · 2012Article
Corrections and comments
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Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
The muscular dystrophies are a heterogeneous group of over 40 disorders that are characterised by muscle weakness and wasting. The most common are Duchenne muscular dystrophy and Becker muscular dystrophy, which result from mutations within the gene encoding dystrophin; myotonic dystrophy type 1, which results from an expanded trinucleotide repeat in the myotonic dystrophy protein kinase gene; and facioscapulohumeral dystrophy, which is associated with contractions in the subtelomeric region of human chromosome 1. Currently the only treatments involve clinical management of symptoms, although several promising experimental strategies are emerging. These include gene therapy using adeno-associated viral, lentiviral and adenoviral vectors and nonviral vectors, such as plasmid DNA. Exon-skipping and cell-based therapies have also shown promise in the effective treatment and regeneration of dystrophic muscle. The availability of numerous animal models for Duchenne muscular dystrophy has enabled extensive testing of a wide range of therapeutic approaches for this type of disorder. Consequently, we focus here on the therapeutic developments for Duchenne muscular dystrophy as a model of the types of approaches being considered for various types of dystrophy. We discuss the advantages and limitations of each therapeutic strategy, as well as prospects and recent successes in the context of future clinical applications.
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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.