ArticleBMC biotechnology2003
In utero recombinant adeno-associated virus gene transfer in mice, rats, and primates.
Article in BMC biotechnology, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 41 citations in OpenAlex.
- Defining a Midgestational Window forbioRxiv : the preprint server for biology · 2026Article
- Targeted gene transfer into developmentally defined cell populations of the primate brain.Cell reports · 2026Article
- Advances in cellular resolution microscopy for brain imaging in rats.Neurophotonics · 2023Review
- Local transgene expression and whole-body transgenesis to model brain diseases in nonhuman primate.Animal models and experimental medicine · 2019Review
- Trisomy silencing by XIST normalizes Down syndrome cell pathogenesis demonstrated for hematopoietic defects in vitro.Nature communications · 2018Article
- In utero lung gene transfer using adeno-associated viral and lentiviral vectors in mice.Human gene therapy methods · 2014Article
- The fetal respiratory system as target for antenatal therapy.Facts, views & vision in ObGyn · 2011Review
- Efficient gene transfer into the mouse lung by fetal intratracheal injection of rAAV2/6.2.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Adult onset lung disease following transient disruption of fetal stretch-induced differentiation.Respiratory research · 2009Article
- Transient in utero disruption of cystic fibrosis transmembrane conductance regulator causes phenotypic changes in alveolar type II cells in adult rats.BMC cell biology · 2009Article
- Anti-metastatic effects of viral and non-viral mediated Nk4 delivery to tumours.Genetic vaccines and therapy · 2009Article
- Small interfering peptide (siP) for in vivo examination of the developing lung interactonome.Developmental dynamics : an official publication of the American Association of Anatomists · 2009Article
- CFTR and Wnt/beta-catenin signaling in lung development.BMC developmental biology · 2008Article
- Efficient in vivo targeting of epidermal stem cells by early gestational intraamniotic injection of lentiviral vector driven by the keratin 5 promoter.Molecular therapy : the journal of the American Society of Gene Therapy · 2008Article
- Angiogenic and antiangiogenic gene therapy.Gene therapy · 2005Review
- Long term physiologic modification using rAAV in utero gene-therapy.Genetic vaccines and therapy · 2004Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundGene transfer into the amniotic fluid using recombinant adenovirus vectors was shown previously to result in high efficiency transfer of transgenes into the lungs and intestines. Adenovirus mediated in utero gene therapy, however, resulted in expression of the transgene for less than 30 days. Recombinant adenovirus associated viruses (rAAV) have the advantage of maintaining the viral genome in daughter cells thus providing for long-term expression of transgenes.
methodsRecombinant AAV2 carrying green fluorescent protein (GFP) was introduced into the amniotic sac of fetal rodents and nonhuman primates. Transgene maintenance and expression was monitor.
resultsGene transfer resulted in rapid uptake and long-term gene expression in mice, rats, and non-human primates. Expression and secretion of the reporter gene, GFP, was readily demonstrated within 72 hours post-therapy. In long-term studies in rats and nonhuman primates, maintenance of GFP DNA, protein expression, and reporter gene secretion was documented for over one year.
conclusionsBecause only multipotential stem cells are present at the time of therapy, these data demonstrated that in utero gene transfer with AAV2 into stem cells resulted in long-term systemic expression of active transgene roducts. Thus, in utero gene transfer via the amniotic fluid may be useful in treatment of gene disorders.
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