ArticleHuman gene therapy2013
Long-term robust myocardial transduction of the dog heart from a peripheral vein by adeno-associated virus serotype-8.
Article in Human gene therapy, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
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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.
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Who cites it
25 citing papers in PubMed, 29 citations in OpenAlex.
- Gene Therapy for Heart Failure: Impact on Mitochondrial Dysfunction.Biomedicines · 2026Review
- Canine Model of Duchenne Muscular Dystrophy.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges.Genes & diseases · 2024Review
- Leverage of nuclease-deficient CasX for preventing pathological angiogenesis.Molecular therapy. Nucleic acids · 2023Article
- Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to Clinical Trials and Back.Journal of personalized medicine · 2020Review
- Systemic Delivery of Adeno-Associated Viral Vectors in Mice and Dogs.Methods in molecular biology (Clifton, N.J.) · 2019Article
- Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients.Human gene therapy · 2018Article
- Nanotherapy for Duchenne muscular dystrophy.Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology · 2018Review
- Use of Adeno-Associated Virus Vector for Cardiac Gene Delivery in Large-Animal Surgical Models of Heart Failure.Human gene therapy. Clinical development · 2017Review
- Adeno-Associated Virus (AAV) as a Vector for Gene Therapy.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2017Review
- Systemic delivery of adeno-associated viral vectors.Current opinion in virology · 2016Review
- Dystrophin Gene Replacement and Gene Repair Therapy for Duchenne Muscular Dystrophy in 2016: An Interview.Human gene therapy. Clinical development · 2016Article
- Characterization and Comparison of Canine Multipotent Stromal Cells Derived from Liver and Bone Marrow.Stem cells and development · 2016Article
- 100-fold but not 50-fold dystrophin overexpression aggravates electrocardiographic defects in the mdx model of Duchenne muscular dystrophy.Molecular therapy. Methods & clinical development · 2016Article
- Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.Human molecular genetics · 2015Article
- Cardiac AAV9 Gene Delivery Strategies in Adult Canines: Assessment by Long-term Serial SPECT Imaging of Sodium Iodide Symporter Expression.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
- AAV-8 is more efficient than AAV-9 in transducing neonatal dog heart.Human gene therapy methods · 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
- Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.Current pediatrics reports · 2014Article
Corrections and comments
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Authors and funding
6 authors at 3 institutions in 3 countries.
Funding
Abstract
Molecular intervention using noninvasive myocardial gene transfer holds great promise for treating heart diseases. Robust cardiac transduction from peripheral vein injection has been achieved in rodents using adeno-associated virus (AAV) serotype-9 (AAV-9). However, a similar approach has failed to transduce the heart in dogs, a commonly used large animal model for heart diseases. To develop an effective noninvasive method to deliver exogenous genes to the dog heart, we employed an AAV-8 vector that expresses human placental alkaline phosphatase reporter gene under the transcriptional regulation of the Rous sarcoma virus promoter. Vectors were delivered to three neonatal dogs at the doses of 1.35×10(14), 7.14×10(14), and 9.06×10(14) viral genome particles/kg body weight via the jugular vein. Transduction efficiency and overall safety were evaluated at 1.5, 2.5, and 12 months postinjection. AAV delivery was well tolerated and dog growth was normal. Blood chemistry and internal organ histology were unremarkable. Widespread skeletal muscle transduction was observed in all dogs without T-cell infiltration. Encouragingly, whole heart myocardial transduction was achieved in two dogs that received higher doses and cardiac expression lasted for at least 1 year. In summary, peripheral vein AAV-8 injection may represent a simple heart gene transfer method in large mammals. Further optimization of this gene delivery strategy may open the door for a readily applicable gene therapy method to treat many heart diseases.
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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.