Cibele Nunes Peroni

• Abstract

  Osteogenesis imperfecta (OI), commonly known as brittle bone disease, is a congenital connective tissue dysplasia, mainly characterized by bone deformity, brittleness and low density, short stature, and other connective tissue changes associated with structural or quantitative changes of collagen. In the case of type I OI, there is a decrease in the amount of type I collagen in the extrac…

  Gene therapy by electrotransfer of mouse growth hormone gene in an animal model of osteogenesis imperfecta, AP.R

• Abstract

  In previous works we have developed alternative methodologies for ex vivo and in vivo gene therapy directed to the phenotypic correction of dwarfism using the human (hGH) and mouse growth hormone (mGH) genes. In these studies two animal models of GH deficiency, the immunocompetent dwarf (lit/lit) and the immunodeficient dwarf (lit/scid) mice, were employed. Both in the ex vivo, based on i…

  Growth hormone gene therapy: plasmid DNA injection and microencapsulated cells implantation in dwarf mice, AP.R
• Evidence for in vivo persistence of mgh-secreting human keratinocytes by immunohistochemical staining of grafted organotypic cultures., AR.BR

• Abstract

  The electrotransfer of plasmid DNA containing the Genes for Human (hGH) or Mouse (mGH) growth hormone has been used by our group. For these studies of in vivo gene therapy, two animal models of GH deficiency, the dwarf (lit/lit) and immunodeficient dwarf (lit/scid) are used. With respect to the hGH gene, sustainable circulating levels of the hormone were obtained during 60 days, and a bod…

  Optimization of in vivo growth hormone gene transfer parameters for phenotypic correction of dwarf mice, BP.MS

• Abstract

  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 approa…

  Growth hormone gene therapy: homologous treatment with plasmid DNA and with implant of microencapsulated cells in immunocompetent dwarf mice (lit/lit), BP.PD

• Abstract

  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 a dwarf (lit/lit) or immunodeficient dwarf (lit/scid) mice. Thus, the secr…

  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, BP.IC

(Only some records are available in English at this moment)

• Abstract

  Gene therapy is a promising treatment modality for inherited and acquired diseases by delivering genes into the target cells to restore or promote specific cellular functions. In spite of the gene therapy potency, the utility of this strategy is defined by the balance between safety and effectiveness. While virus-derived vectors have a high efficiency in the DNA deliver in comparison with…

  Gene therapy for growth hormone deficiency mediated by minicircle DNA electrotransfer in dwarf mice, BE.EP.DR

• Abstract

  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 preclinica…

  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