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Growth Hormone Gene therapy: homologous treatment based on plasmid DNA administration and on microencapsulated cells implanted in immunocompetent dwarf mice (lit/lit)

Grant number: 14/04277-0
Support type:Scholarships abroad - Research Internship - Post-doctor
Effective date (Start): April 01, 2014
Effective date (End): March 31, 2015
Field of knowledge:Biological Sciences - Biochemistry
Principal Investigator:Cibele Nunes Peroni
Grantee:Cláudia Regina Cecchi
Supervisor abroad: Lars Aagaard
Home Institution: Instituto de Pesquisas Energéticas e Nucleares (IPEN). Secretaria de Desenvolvimento Econômico (São Paulo - Estado). São Paulo , SP, Brazil
Local de pesquisa : Aarhus University, Denmark  
Associated to the scholarship:13/03747-0 - Growth Hormone Gene Therapy: homologous treatment with plasmid DNA and with implant of microencapsulated cells in immunocompetent dwarf mice (lit/lit), BP.PD


In previous work we have demonstrated significant phenotypic effects in immunodeficient mice dwarfs obtained upon administration of plasmid DNA containing the growth hormone gene (GH) , followed by electroporation . These effects were also comparable to those obtained by repeated injections of recombinant hormone in the same animals . In this project , in order to get closer to preclinical models , we aim to: a) continue the research already undertaken in relation to homologous models of gene therapy in immunocompetent mice that were treated with the mouse GH gene (mGH ) compared with human (hGH ), B) to implant in the same lit/lit mice human retinal microencapsulated epithelial cells, transfected by a transposon and expressing hGH or mGH. In the first case , based on the data already submitted by the candidate ( PhD thesis ) electroporation protocol modified to do not exposure the muscle, using a combination of high and low pulses. Also the construction of vectors that enable greater levels of circulating IGF-I, in analogy to the conditions for treatment to children with GH deficiency. In the second case an alternative technique, where human epithelial cells already utilized in clinical cell therapy protocols will be used for the first time in a model to the treatment of GH deficiency will be developed. (AU)

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.

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