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Growth Hormone Gene Therapy: homologous treatment with plasmid DNA and with implant of microencapsulated cells in immunocompetent dwarf mice (lit/lit)

Grant number: 13/03747-0
Support type:Scholarships in Brazil - Post-Doctorate
Effective date (Start): September 01, 2013
Effective date (End): September 24, 2016
Field of knowledge:Biological Sciences - Biochemistry
Principal Investigator:Cibele Nunes Peroni
Grantee:Cláudia Regina Cecchi
Home Institution: Instituto de Pesquisas Energéticas e Nucleares (IPEN). Secretaria de Desenvolvimento Econômico (São Paulo - Estado). São Paulo , SP, Brazil
Associated scholarship(s):14/04277-0 - Growth Hormone Gene therapy: homologous treatment based on plasmid DNA administration and on microencapsulated cells implanted in immunocompetent dwarf mice (lit/lit), BE.EP.PD


In previous work we already demonstrated important phenotypic effects obtained in immunodeficient dwarf mice via administration of plasmid DNA containing the growth hormone (GH) gene, followed by electroporation. These effects were also found comparable to those obtained via injections of repeated recombinant hormone in the same animal model. In the present project, trying a better approach to pre-clinical testing, we plan: A) to give continuity to the researches already in progress about an homologous model of gene therapy in which immunocompetent mice are being treated with the gene of mice GH (mGH), in comparison with the human (hGH); B) to implant human retinal microencapsulated epithelial cells, transfected by a transposon and expressing hGH or mGH, also in lit/lit mice. In the first case, starting from data already obtained by the candidate (Ph.D. thesis) and that are greatly in favor of the utilization of mGH, we will develop gene therapy models in younger mice (30-40 days of age), also using vectors and conditions that allow higher levels of circulating IGF-I, in analogy with the treatment conditions of GH-deficient children. In the second case we will develop an alternative technique, in which human epithelial cells already quite used in clinical protocols of cell therapy, will be used for the first time in a model for the treatment of GH deficiency.

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, C. R.; HIGUTI, E.; LIMA, E. R.; VIEIRA, D. P.; SQUAIR, P. L.; PERONI, C. N.; BARTOLINI, P. Efficient Non-Invasive Plasmid-DNA Administration into Tibialis Cranialis Muscle of ``Little{''} Mice. CURRENT MOLECULAR MEDICINE, v. 17, n. 3, p. 230-235, 2017. Web of Science Citations: 0.
HIGUTI, ELIZA; CECCHI, CLAUDIA R.; OLIVEIRA, NELIO A. J.; LIMA, ELIANA R.; VIEIRA, DANIEL P.; AAGAARD, LARS; JENSEN, THOMAS G.; JORGE, ALEXANDER A. L.; BARTOLINI, PAOLO; PERONI, CIBELE N. Partial correction of the dwarf phenotype by non-viral transfer of the growth hormone gene in mice: Treatment age is critical. GROWTH HORMONE & IGF RESEARCH, v. 26, p. 1-7, FEB 2016. Web of Science Citations: 2.
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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