ReviewGene therapy2004
Liver gene therapy: advances and hurdles.
Review in Gene therapy, 2004. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
27 citing papers in PubMed.
- Review
- Review
- Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia.PloS one · 2022Article
- AAV serotype 8-mediated liver specific GNMT expression delays progression of hepatocellular carcinoma and prevents carbon tetrachloride-induced liver damage.Scientific reports · 2018Article
- Long-Term Correction of Diabetes in Mice by In Vivo Reprogramming of Pancreatic Ducts.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Induction of apoptosis in imatinib sensitive and resistant chronic myeloid leukemia cells by efficient disruption of bcr-abl oncogene with zinc finger nucleases.Journal of experimental & clinical cancer research : CR · 2018Article
- Alternative Start Sites Downstream of Non-Sense Mutations Drive Antigen Presentation and Tolerance Induction to C-Terminal Epitopes.Journal of immunology (Baltimore, Md. : 1950) · 2017Article
- Charge-altering releasable transporters (CARTs) for the delivery and release of mRNA in living animals.Proceedings of the National Academy of Sciences of the United States of America · 2017Article
- Hereditary Angioedema as a Metabolic Liver Disorder: Novel Therapeutic Options and Prospects for Cure.Frontiers in immunology · 2016Article
- Phosphorylation of the nuclear receptor corepressor 1 by protein kinase B switches its corepressor targets in the liver in mice.Hepatology (Baltimore, Md.) · 2015Article
- Therapeutic genome editing: prospects and challenges.Nature medicine · 2015Review
- Therapeutic efficiency of folated poly(ethylene glycol)-chitosan-graft-polyethylenimine-Pdcd4 complexes in H-ras12V mice with liver cancer.International journal of nanomedicine · 2013Article
- The liver as a target organ for gene therapy: state of the art, challenges, and future perspectives.Pharmaceuticals (Basel, Switzerland) · 2012Article
- Gene transfer of c-met confers protection against D-galactosamine/lipopolysaccharide-induced acute liver failure.Digestive diseases and sciences · 2012Article
- Design and synthesis of dual-ligand modified chitosan as a liver targeting vector.Journal of materials science. Materials in medicine · 2012Article
- Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk.Hepatology (Baltimore, Md.) · 2011Article
- Intra-arterial targeted islet-specific expression of Sirt1 protects β cells from streptozotocin-induced apoptosis in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Gene therapy for liver regeneration: experimental studies and prospects for clinical trials.World journal of gastroenterology · 2010Review
- Gene therapy for dyslipidemia: a review of gene replacement and gene inhibition strategies.Clinical lipidology · 2010Article
- AAV8-mediated gene therapy prevents induced biochemical attacks of acute intermittent porphyria and improves neuromotor function.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Liver gene therapy is being developed as an alternative to orthotopic liver transplantation, which is the only effective therapy for many liver diseases. The liver has unique features that make it attractive for in vivo and ex vivo gene transfer. In vivo approach is far less invasive than ex vivo approach, although in most cases, host immune response directed against the transgene product and/or vector particles severely impairs the efficiency of gene transfer, and precludes long-term transgene expression after in vivo gene delivery. Ex vivo approach allows for an elective targeting of the hepatocytes, avoiding that the transgene be expressed in professional antigen-presenting, but is faced with the low in vitro proliferative ability of hepatocytes, and to the low in vivo liver repopulating ability of transplanted cells. In some specific situations where immune response was controlled or transplanted cells had a strong growth advantage over host hepatocytes, gene transfer resulted in long-term and complete correction of a liver genetic defect. In this review, we will outline the liver diseases that may benefit from gene therapy, the vector technology under investigation, the advances and the problems to be overcome.
Indexed as
Identifiers
15454961What 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.