ReviewILAR journal2009
Gene therapy in large animal models of muscular dystrophy.
Review in ILAR journal, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
23 citing papers in PubMed, 60 citations in OpenAlex.
- Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side.Pharmaceutics · 2020Review
- Effective restoration of dystrophin expression in iPSCComputational and structural biotechnology journal · 2020Article
- Gene therapies in canine models for Duchenne muscular dystrophy.Human genetics · 2019Review
- 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
- Myelination is delayed during postnatal brain development in the mdx mouse model of Duchenne muscular dystrophy.BMC neuroscience · 2017Article
- Article
- Multiple Species Comparison of Cardiac Troponin T and Dystrophin: Unravelling the DNA behind Dilated Cardiomyopathy.Journal of cardiovascular development and disease · 2017Review
- Use of Adeno-Associated Virus to Enrich Cardiomyocytes Derived from Human Stem Cells.Human gene therapy. Clinical development · 2015Article
- A COLQ Missense Mutation in Sphynx and Devon Rex Cats with Congenital Myasthenic Syndrome.PloS one · 2015Article
- Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Impacts of dystrophin and utrophin domains on actin structural dynamics: implications for therapeutic design.Journal of molecular biology · 2012Article
- Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- The correlation analysis of functional factors and age with duchenne muscular dystrophy.Annals of rehabilitation medicine · 2012Article
- Long-term engraftment of multipotent mesenchymal stromal cells that differentiate to form myogenic cells in dogs with Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Long-term systemic myostatin inhibition via liver-targeted gene transfer in golden retriever muscular dystrophy.Human gene therapy · 2011Article
- Dog models of naturally occurring cancer.Trends in molecular medicine · 2011Review
- Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model.Human gene therapy · 2011Article
- Immune Responses to rAAV6: The Influence of Canine Parvovirus Vaccination and Neonatal Administration of Viral Vector.Frontiers in microbiology · 2011Article
- Immune responses to AAV in canine muscle monitored by cellular assays and noninvasive imaging.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Man's best friend becomes biology's best in show: genome analyses in the domestic dog.Annual review of genetics · 2010Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 1 country.
Funding
Abstract
The muscular dystrophies are a group of genetically and phenotypically heterogeneously inherited diseases characterized by progressive muscle wasting, which can lead to premature death in severe forms such as Duchenne muscular dystrophy (DMD). In many cases they are caused by the absence of proteins that are critical components of the dystrophin-glycoprotein complex, which links the cytoskeleton and the basal lamina. There is no effective treatment for these disorders at present, but several novel strategies for replacing or repairing the defective gene are in development, with early encouraging results from animal models. We review these strategies, which include the use of stem cells of different tissue origins, gene replacement therapies mediated by various viral vectors, and transcript repair treatments using exon skipping strategies. We comment on their advantages and on limitations that must be overcome before successful application to human patients. Our focus is on studies in a clinically relevant large canine model of DMD. Recent advances in the field suggest that effective therapies for muscular dystrophies are on the horizon. Because of the complex nature of these diseases, it may be necessary to combine multiple approaches to achieve a successful treatment.
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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.