ArticleMolecular therapy : the journal of the American Society of Gene Therapy2012
Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.
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Who cites it
33 citing papers in PubMed, 52 citations in OpenAlex.
- Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression.The Journal of clinical investigation · 2013Trial
- Polyplex Nanomicelle-Mediated Pgc-1α4 mRNA Delivery Via Hydrodynamic Limb Vein Injection Enhances Damage Resistance in Duchenne Muscular Dystrophy Mice.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Delivery of genetic medicines for muscular dystrophies.Cell reports. Medicine · 2025Review
- Comprehensive Evaluation of Lipid Nanoparticles and Polyplex Nanomicelles for Muscle-Targeted mRNA Delivery.Pharmaceutics · 2023Article
- Selective attachment of a microtubule interacting peptide to plasmid DNA via a triplex forming oligonucleotide for transfection improvement.Gene therapy · 2023Article
- Safety levels of systemic IL-12 induced by cDNA expression as a cancer therapeutic.Immunotherapy · 2022Article
- DNA nuclear targeting sequences for enhanced non-viral gene transfer: AnMolecular therapy. Nucleic acids · 2021Article
- AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation.Molecular therapy. Methods & clinical development · 2021Article
- Plasmid-Mediated Gene Therapy in Mouse Models of Limb Girdle Muscular Dystrophy.Molecular therapy. Methods & clinical development · 2019Article
- Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity.Human gene therapy · 2019Article
- Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study.Molecular therapy. Methods & clinical development · 2019Article
- Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model.Blood advances · 2018Article
- DNA-Mediated Gene Therapy in a Mouse Model of Limb Girdle Muscular Dystrophy 2B.Molecular therapy. Methods & clinical development · 2017Article
- Long-term effects of systemic gene therapy in a canine model of myotubular myopathy.Muscle & nerve · 2017Article
- Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.Nature communications · 2017Article
- The golden retriever model of Duchenne muscular dystrophy.Skeletal muscle · 2017Review
- Gene therapy in monogenic congenital myopathies.Methods (San Diego, Calif.) · 2016Review
- Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods.Human gene therapy. Clinical development · 2015Review
- High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline.Human gene therapy · 2015Article
- Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency.Human gene therapy methods · 2015Review
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
Abstract
We evaluated safety and feasibility of the transvenous limb perfusion gene delivery method in muscular dystrophy. A dose escalation study of single limb perfusion with 0.9% saline starting with 5% of limb volume was carried out in adults with muscular dystrophies under intravenous analgesia/anesthesia. Cardiac, vascular, renal, muscle, and nerve functions were monitored. A tourniquet was placed above the knee with inflated pressure of 310 mm Hg. Infusion was carried out with a clinically approved infuser via an intravenous catheter inserted in the saphenous vein with a goal infusion rate of 80 ml/minute. Infusion volume was escalated stepwise to 20% limb volume in seven subjects. No subject complained of any post procedure pain other than due to needle punctures. Safety warning boundaries were exceeded only for transient depression of limb tissue oximetry and transient elevation of muscle compartment pressures; these were not associated with nerve, muscle, or vascular damage. Muscle magnetic resonant imaging (MRI) demonstrated fluid accumulation in muscles of the perfused lower extremity. High-pressure retrograde transvenous limb perfusion with saline up to 20% of limb volume at above infusion parameters is safe and feasible in adult human muscular dystrophy. This study will serve as a basis for future gene transfer clinical trials.
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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.