Diseño y caracterización molecular y funcional de virus artificiales y nanopartículas de arquitectura modular para el transporte dirigido de DNA terapéutico

Project Details

Description

The continuous identification of genetic pathologies as well as of the molecular basis of diverse degenerative, inflammatory and infectious diseases allows to take peculiar biomedical approaches of health base don the selective and/or directed administration of therapeutic DNA or RNA, expressible or not. Nevertheless, the strategies of directed administration must be biologically safe, guarantee the stability of the transported material, their proper biodistribution and targeted delivery to the cell cytoplasm or nucleus, with phenotypical significant effectiveness. The most conventional vectors are based on genetically modified viruses, in spite of representing important biological concerns for the present and foreseeable future safety requirements. For that reason, there is a wide consensus on the need to explore new non-viral vehicles that being able to mimic properties of these cellular parasites lack their biological risks. The present project proposes the application of modular protein design strategies, through the correct combination of functional domains, to the generation of a catalogue of prototypes of interest as gene therapy vehicles, based solely on single-chain, multifunctional polypeptides. The protein design strategies will be based on the previous experience of our group, and the resulting vehicles will be characterized quantitatively through a range of properties that will include productivity, purification, self-assembling, stability, specificity, toxicity, endosomal escape, nuclear transport, proper uncoating and gene expression. The structure of the project is essentially horizontal or comparative (&)
StatusFinished
Effective start/end date1/12/0730/11/10

Funding

  • Ministerio de Educación y Ciencia: €147,620.00

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