ArticleBlood2005
Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer.
Article in Blood, 2005. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 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
31 citing papers in PubMed, 76 citations in OpenAlex.
- Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy.Molecular therapy. Methods & clinical development · 2019Review
- Long-term correction of hemophilia A mice following lentiviral mediated delivery of an optimized canine factor VIII gene.Gene therapy · 2017Article
- Lentiviral Vectors Pseudotyped with Filoviral Glycoproteins.Methods in molecular biology (Clifton, N.J.) · 2017Article
- Gene therapy for hemophilia.Frontiers in bioscience (Landmark edition) · 2015Review
- Delivery of full-length factor VIII using a piggyBac transposon vector to correct a mouse model of hemophilia A.PloS one · 2014Article
- piggyBac-mediated phenotypic correction of factor VIII deficiency.Molecular therapy. Methods & clinical development · 2014Article
- A TLR and non-TLR mediated innate response to lentiviruses restricts hepatocyte entry and can be ameliorated by pharmacological blockade.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- A Hyperactive Transposase Promotes Persistent Gene Transfer of a piggyBac DNA Transposon.Molecular therapy. Nucleic acids · 2012Article
- Advances in Overcoming Immune Responses following Hemophilia Gene Therapy.Journal of genetic syndromes & gene therapy · 2011Article
- Rational design, fabrication, characterization and in vitro testing of biodegradable microparticles that generate targeted and sustained transgene expression in HepG2 liver cells.Journal of drug targeting · 2011Article
- Genetic engineering of murine CD8+ and CD4+ T cells for preclinical adoptive immunotherapy studies.Journal of immunotherapy (Hagerstown, Md. : 1997) · 2011Article
- A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Cell entry of enveloped viruses.Advances in genetics · 2011Review
- Perinatal gene transfer to the liver.Current pharmaceutical design · 2011Review
- Manipulating Immune Tolerance with Micro-RNA Regulated Gene Therapy.Frontiers in microbiology · 2011Article
- Development and Applications of VSV Vectors Based on Cell Tropism.Frontiers in microbiology · 2011Article
- Acquisition of complement resistance through incorporation of CD55/decay-accelerating factor into viral particles bearing baculovirus GP64.Journal of virology · 2010Article
- Recent advances in lentiviral vector development and applications.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Review
- Delivery of factor VIII gene into skeletal muscle cells using lentiviral vector.Yonsei medical journal · 2010Article
- The role of liver sinusoidal cells in hepatocyte-directed gene transfer.The American journal of pathology · 2010Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 1 institution in 1 country.
Funding
Abstract
Hemophilia A is a clinically important coagulation disorder caused by the lack or abnormality of plasma coagulation factor VIII (FVIII). Gene transfer of the FVIII cDNA to hepatocytes using lentiviral vectors is a potential therapeutic approach. We investigated the efficacy of feline immunodeficiency virus (FIV)-based vectors in targeting hepatocytes and correcting FVIII deficiency in a hemophilia A mouse model. Several viral envelope glycoproteins were screened for efficient FIV vector pseudotyping and hepatocyte transduction. The GP64 glycoprotein from baculovirus Autographa californica multinuclear polyhedrosis virus pseudo-typed FIV efficiently and showed excellent hepatocyte tropism. The GP64-pseudotyped vector was stable in the presence of human or mouse complement. Inclusion of a hybrid liver-specific promoter (murine albumin enhancer/human alpha1-antitrypsin promoter) further enhanced transgene expression in hepatocytes. We generated a GP64-pseudotyped FIV vector encoding the B domain-deleted human FVIII coding region driven by the liver-specific promoter, with 2 beneficial point mutations in the A1 domain. Intravenous vector administration conferred sustained FVIII expression in hemophilia A mice for several months without the generation of anti-human FVIII antibodies and resulted in partial phenotypic correction. These findings demonstrate the utility of GP64-pseudotyped FIV lentiviral vectors for targeting hepatocytes to correct disorders associated with deficiencies of secreted proteins.
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What OpenQuestion holds
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.