Desenvolupament d'adenovirus integratius Gutless Helper-Free com vectors de teràpia gènica. Noves estratègies basades en la utilització conjunta del transposon humà HSMAR2 i la recombinasa PHIC

  • Chillón Rodriguez, Miguel (Principal Investigator)
  • Alba Fernández, Raul (Scholar)
  • Monfar Rebull, Maria Mercè (Researcher on contract)

Project Details


Integrative gutless adenovirus vectors are very attractive for gene therapy because they do not induce activation of the immune response since they carry no viral genes in their genome, and also allow integration of the therapeutic gene in the genome of the target cell. To generate these vectors we will use the unidirectional C31 recombinase, as well as the human mariner transposon Hsmar2, which has been recently generically reconstructed by us. Compared to other gutless or integrative adenovirus the choice of these systems means for the first time, the avoidance of contamination with the helper virus, and also the minimization of the effect of specie-specific nuclear factors since Hsmar2 comes from human genome and not from Salmonids or invertebrates. These advantages will allow to produce GMP-quality integrative gutless adenovirus to be used in human clinical trials, which is not possible now with the current technology. Newly generated integrative gutless adenovirus will be characterized in vitro to determine the absence of contaminat helper virus and to analyze specificity and efficiency of integration. Then, the vectors will be administered in vivo to different organs to analyze both its ability to induce the host immune response and, its efficiency concerning duration and expression levels of the therapeutic genes
Effective start/end date15/12/0314/12/06


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