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Electrotransfer of a plasmid containing the gene for murine growth hormone in dwarf mice to the obtainment of a homologous model of in vivo gene therapy.

Grant number: 11/23365-9
Support type:Scholarships in Brazil - Scientific Initiation
Effective date (Start): April 01, 2012
Effective date (End): December 31, 2013
Field of knowledge:Biological Sciences - Biochemistry - Molecular Biology
Principal Investigator:Cibele Nunes Peroni
Grantee:Eliana Rosa Lima Filha
Home Institution: Instituto de Pesquisas Energéticas e Nucleares (IPEN). Secretaria de Desenvolvimento Econômico (São Paulo - Estado). São Paulo , SP, Brazil


In our laboratory, a research line in the field of gene therapy for deficiency of growth hormone (GH) is developed. Among the strategies studied, one of them is based on a non-viral technique of direct injection of plasmid DNA (naked DNA), associated with the electrotransfer, using intramuscular administration into dwarf (lit/lit) or immunodeficient dwarf (lit/scid) mice. Thus, the secretion of human growth hormone (hGH) was for the first time maintained for a period of 60 days in the circulation of lit/scid mice, with circulating levels from 1.5 to 3.0 ng hGH/mL followed by a significant weight gain in these treated animals, approximately 30%, compared to the control group (saline injection followed by electroporation). In this project, the goal is to use a homologous system following the electrotransfer of a plasmid containing the gene of the mouse growth hormone (mGH) into dwarf mice (lit/lit). With this model, it is expected to mimic the response that would be obtained in GH-deficient individuals, i.e., administration of a homologous gene into a immunocompetent organism. Please note that this activity is part of a research project recently approved by FAPESP (2010/51116-0).

Scientific publications
(References retrieved automatically from Web of Science and SciELO through information on FAPESP grants and their corresponding numbers as mentioned in the publications by the authors)
CECCHI, CLAUDIA R.; HIGUTI, ELIZA; OLIVEIRA, NELIO A. J.; LIMA, ELIANA R.; JAKOBSEN, MARIA; DAGNAES-HANSEN, FREDERICK; GISSEL, HANNE; AAGAARD, LARS; JENSEN, THOMAS G.; JORGE, ALEXANDER A. L.; BARTOLINI, PAOLO; PERONI, CIBELE N. A Novel Homologous Model for Gene Therapy of Dwarfism by Non-Viral Transfer of the Mouse Growth Hormone Gene into Immunocompetent Dwarf Mice. CURRENT GENE THERAPY, v. 14, n. 1, p. 44-51, FEB 2014. Web of Science Citations: 6.

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