ArticlePharmaceuticals (Basel, Switzerland)2013
Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.
Article in Pharmaceuticals (Basel, Switzerland), 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 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
21 citing papers in PubMed.
- Navigating the Hurdles of Intra-Articular AAV Gene Therapy.International journal of molecular sciences · 2025Review
- Preferences for gene therapy in Duchenne muscular dystrophy: insights from patient and caregiver interviews and attribute development.Frontiers in pharmacology · 2025Article
- Dental-Pulp Stem Cells as a Therapeutic Strategy for Ischemic Stroke.Biomedicines · 2022Review
- rAAV8 and rAAV9-Mediated Long-Term Muscle Transduction with Tacrolimus (FK506) in Non-Human Primates.Molecular therapy. Methods & clinical development · 2020Article
- Cell-based interferon gene therapy using proliferation-controllable, interferon-releasing mesenchymal stem cells.Scientific reports · 2019Article
- 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
- Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy.Journal of personalized medicine · 2019Review
- Muscle Wasting Diseases: Novel Targets and Treatments.Annual review of pharmacology and toxicology · 2019Review
- Bone-Targeted Alkaline Phosphatase Treatment of Mandibular Bone and Teeth in Lethal Hypophosphatasia via an scAAV8 Vector.Molecular therapy. Methods & clinical development · 2018Article
- Current and Emerging Therapies for Duchenne Muscular Dystrophy.Current treatment options in neurology · 2018Review
- Genome Editing in Induced Pluripotent Stem Cells using CRISPR/Cas9.Stem cell reviews and reports · 2018Review
- Chemotherapeutics overcoming nonsense mutation-associated genetic diseases: medicinal chemistry of negamycin.The Journal of antibiotics · 2018Review
- Myopenia and precision (P4) medicine.Journal of cachexia, sarcopenia and muscle · 2017Article
- Structure-Activity Relationship Study of Leucyl-3-ACS medicinal chemistry letters · 2017Article
- Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy.Journal of human genetics · 2017Review
- Current Translational Research and Murine Models For Duchenne Muscular Dystrophy.Journal of neuromuscular diseases · 2016Review
- Current and emerging treatment strategies for Duchenne muscular dystrophy.Neuropsychiatric disease and treatment · 2016Review
- Treatment of hypophosphatasia by muscle-directed expression of bone-targeted alkaline phosphatase via self-complementary AAV8 vector.Molecular therapy. Methods & clinical development · 2016Article
- X-Linked Dilated Cardiomyopathy: A Cardiospecific Phenotype of Dystrophinopathy.Pharmaceuticals (Basel, Switzerland) · 2015Review
- Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9.Stem cell reports · 2015Article
Corrections and comments
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Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Various characteristics of adeno-associated virus (AAV)-based vectors with long-term safe expression have made it an exciting transduction tool for clinical gene therapy of Duchenne muscular dystrophy (DMD). Although host immune reactions against the vector as well as transgene products were detected in some instances of the clinical studies, there have been promising observations. Methods of producing AAV vectors for considerable in vivo experimentation and clinical investigations have been developed and a number of studies with AAV vector-mediated muscle transduction were attempted. Notably, an intravenous limb perfusion transduction technique enables extensive transgene expression in the skeletal muscles without noticeable adverse events. Furthermore, cardiac transduction by the rAAV9-microdystrophin would be promising to prevent development of cardiac dysfunction. Recent achievements in transduction technology suggest that long-term transgene expression with therapeutic benefits in DMD treatment would be achieved by the rAAV-mediated transduction strategy with an adequate regimen to regulate host immune response.
Identifiers
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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.