ArticleGene therapy2003
Long-term therapeutic levels of human alpha-1 antitrypsin in plasma after hydrodynamic injection of nonviral DNA.
Article in Gene therapy, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 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
38 citing papers in PubMed, 83 citations in OpenAlex.
- Hydrodynamic Delivery of IL-10 Gene for Local Immunomodulation in Human Crohn's Disease Tissue: A Proof-of-Concept Study.Pharmaceutics · 2026Article
- Review
- Safe Procedure for Efficient Hydrodynamic Gene Transfer to Isolated Porcine Liver in Transplantation.International journal of molecular sciences · 2024Article
- CMV/AAT promoter of MAR-based episomal vector enhanced transgene expression in human hepatic cells.3 Biotech · 2023Article
- Early Subcellular Hepatocellular Alterations in Mice Post Hydrodynamic Transfection: An Explorative Study.Cancers · 2023Article
- Gene Therapy in Rare Respiratory Diseases: What Have We Learned So Far?Journal of clinical medicine · 2020Review
- Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Review
- Alpha-1 antitrypsin deficiency: outstanding questions and future directions.Orphanet journal of rare diseases · 2018Review
- Review
- Functional Rescue of ABCC6 Deficiency by 4-Phenylbutyrate Therapy Reduces Dystrophic Calcification in Abcc6The Journal of investigative dermatology · 2017Article
- Technical Improvement and Application of Hydrodynamic Gene Delivery in Study of Liver Diseases.Frontiers in pharmacology · 2017Review
- Gene Therapy for Alpha-1 Antitrypsin Deficiency Lung Disease.Annals of the American Thoracic Society · 2016Review
- Human AAT gene transfer to pig liver improved by using a perfusion isolated organ endovascular procedure.European radiology · 2016Article
- Studying Closed Hydrodynamic Models of "In Vivo" DNA Perfusion in Pig Liver for Gene Therapy Translation to Humans.PloS one · 2016Article
- Site-Specific Impact of a Regional Hydrodynamic Injection: Computed Tomography Study during Hydrodynamic Injection Targeting the Swine Liver.Pharmaceutics · 2015Article
- Optimized human factor IX expression cassettes for hepatic-directed gene therapy of hemophilia B.Frontiers of medicine · 2015Article
- Durable expression of minicircle DNA-liposome-delivered androgen receptor cDNA in mice with hepatocellular carcinoma.BioMed research international · 2014Article
- Parameters Affecting Image-guided, Hydrodynamic Gene Delivery to Swine Liver.Molecular therapy. Nucleic acids · 2013Article
- DNA delivery to 'ex vivo' human liver segments.Gene therapy · 2012Article
- Hydrodynamic gene delivery and its applications in pharmaceutical research.Pharmaceutical research · 2011Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The transfection efficacy of several vectors containing the full genomic hAAT gene with its natural promoter (pTG7101) and others containing the cDNA of hAAT gene driven by cytomegalovirus immediate-early promoter or the 0.5 kb upstream of hAAT gene sequence has been studied by hydrodynamic tail-vein injection (20 microg/mouse). pTG7101 (but not the other plasmids) results in therapeutic and stable concentration of hAAT in plasma. A dose-response study with this plasmid (0.3-320 microg/mouse) confirms that hAAT remains long-term stable in plasma, with therapeutic concentrations of hAAT (>0.9 mg/ml). The parameters of the dose-response curve were: R: 0.98, E(max) 3449.0+/- 279.7 microg/ml and EC(50) 1.2 x 10(12) plasmid-gene units. In addition, 4 months after transfection, the intrinsic efficacy of transgenic expression (amount of RNA/DNA) in mouse liver was 50-80% that normally expressed by the mouse gene. The important efficacy of nonviral genomic DNA opens a new avenue in the safety applications of human gene therapy.
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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.