ArticleGene therapy1999
Successful expression of beta-galactosidase and factor IX transgenes in fetal and neonatal sheep after ultrasound-guided percutaneous adenovirus vector administration into the umbilical vein.
Article in Gene therapy, 1999. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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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.
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Who cites it
13 citing papers in PubMed, 76 citations in OpenAlex.
- Gene modification therapies for hereditary diseases in the fetus.Prenatal diagnosis · 2023Review
- Mechanistic Insights into Factor VIII Immune Tolerance Induction via Prenatal Cell Therapy in Hemophilia A.Current stem cell reports · 2019Article
- In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application.Molecular therapy. Methods & clinical development · 2016Review
- Hemophilia A: an ideal disease to correct in utero.Frontiers in pharmacology · 2014Review
- Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems.Gene therapy · 2013Article
- Article
- Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene Delivery.Journal of genetic syndromes & gene therapy · 2012Article
- Factors determining the risk of inadvertent retroviral transduction of male germ cells after in utero gene transfer in sheep.Human gene therapy · 2009Article
- Evaluation of prenatal intra-amniotic LAMB3 gene delivery in a mouse model of Herlitz disease.Gene therapy · 2006Article
- Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice.Gene therapy · 2003Article
- Possible mechanism of gene transfer into early to mid-gestational mouse fetuses by tail vein injection.Gene therapy · 2002Article
- On the scientific and ethical issues of fetal somatic gene therapy.Gene therapy · 2002Article
- Fetal gene therapy.Gene therapy · 1999Article
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Authors and funding
11 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In utero somatic gene therapy in the later stages of pregnancy may allow targeting of organ systems which are difficult to reach later in life and to prevent the development of tissue damage otherwise caused by the early onset of inherited diseases. We report here on the percutaneous delivery of two adenoviral vectors, containing the beta-galactosidase reporter gene and the human Factor IX gene respectively, to the fetal liver and circulation by ultrasound-guided umbilical vein puncture similar to procedures used in human pregnancy. Vector spread, as detected by PCR analysis for the beta-galactosidase encoding vector, was found in almost all fetal and neonatal organs and in the maternal liver. Expression of the beta-galactosidase transgene was detected in many fetal tissues by RT-PCR. High beta-galactosidase production was shown by immuno-histochemistry predominantly in the liver, where about 30percent of the hepatocytes stained positive, and in the adrenal cortex. Production of factor IX was determined by ELISA in the plasma of treated fetuses and newborn lambs and reached at birth up to 80percent of the normal human plasma concentration. This demonstrates a very hopeful proof of principle for the development of prenatal treatment of many genetic diseases but also requires more detailed investigations with respect to the observed systemic spread of the vector.
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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.