ArticleHuman gene therapy2010
Evaluation of hydrodynamic limb vein injections in nonhuman primates.
Article in Human gene therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
13 citing papers in PubMed.
- Selective attachment of a microtubule interacting peptide to plasmid DNA via a triplex forming oligonucleotide for transfection improvement.Gene therapy · 2023Article
- DNA nuclear targeting sequences for enhanced non-viral gene transfer: AnMolecular therapy. Nucleic acids · 2021Article
- Plasmid-Mediated Gene Therapy in Mouse Models of Limb Girdle Muscular Dystrophy.Molecular therapy. Methods & clinical development · 2019Article
- DNA-Mediated Gene Therapy in a Mouse Model of Limb Girdle Muscular Dystrophy 2B.Molecular therapy. Methods & clinical development · 2017Article
- A targeted controlled force injection of genetic material in vivo.Molecular therapy. Methods & clinical development · 2016Article
- High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline.Human gene therapy · 2015Article
- Gene therapy for muscular dystrophy: moving the field forward.Pediatric neurology · 2014Review
- Condensation of plasmid DNA enhances mitochondrial association in skeletal muscle following hydrodynamic limb vein injection.Pharmaceuticals (Basel, Switzerland) · 2014Article
- Autophagy as a new therapeutic target in Duchenne muscular dystrophy.Cell death & disease · 2012Article
- Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Dose response in rodents and nonhuman primates after hydrodynamic limb vein delivery of naked plasmid DNA.Human gene therapy · 2011Article
- Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
- Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease.Gene therapy · 2010Article
Corrections and comments
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Authors and funding
9 authors.
Funding
Abstract
The administration route is emerging as a critical aspect of nonviral and viral vector delivery to muscle, so as to enable gene therapy for disorders such as muscular dystrophy. Although direct intramuscular routes were used initially, intravascular routes are garnering interest because of their ability to target multiple muscles at once and to increase the efficiency of delivery and expression. For the delivery of naked plasmid DNA, our group has developed a hydrodynamic, limb vein procedure that entails placing a tourniquet over the proximal part of the target limb to block all blood flow and injecting the gene vector rapidly in a large volume so as to enable the gene vector to be extravasated and to access the myofibers. The present study was conducted in part to optimize the procedure in preparation for a human clinical study. Various injection parameters such as the effect of papaverine preinjection, tourniquet inflation pressure and duration, and rate of injection were evaluated in rats and nonhuman primates. In addition, the safety of the procedure was further established by determining the effect of the procedure on the neuromuscular and vascular systems. The results from these studies provide additional evidence that the procedure is well tolerated and they provide a foundation on which to formulate the procedure for a human clinical study.
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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.