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Strategic lines of research of the programme BM 1. GENE THERAPY AND CELL THERAPY:
• Stem and progenitor cells . Cellular reprogramming . • Development of non-viral vectors for gene therapy .
Gene therapy and cell therapy are overlapping fields of biomedical research with the goals of repairing the direct cause of genetic diseases in the DNA or cellular population, respectively .
These powerful strategies are also being focused on modulating specific genes and cell subpopulations in acquired diseases in order to re-establish the normal balance . In many diseases, gene and cell therapy are combined in the development of promising therapies .
In addition, these two fields have helped provide reagents, concepts, and techniques that are elucidating the finer points of gene regulation, stem cell lineage, cell-cell interactions, feedback loops, amplification loops, regenerative capacity, and remodelling .
Specifically, Gene therapy is defined as a set of strategies that modify the expression of an individual’s genes or that correct abnormal genes . Each strategy involves the administration of a specific DNA (or RNA) .
Within this framework, CIBER-BBN’s research groups are focused on finding an appropriate use of this novel strategy to deliver new and improved therapies . Viral gene transfer is relatively efficient but there are con- cerns relating to the use of viral vectors in humans . Conversely, non-viral vectors appear safe but inefficient . Therefore, the development of an efficient non-viral vector remains a highly desirable goal and has been recently adopted as part of CIBER-BBN’s Research Programme .
On the other hand, Cell therapy is defined as the administration of live whole cells or maturation of a spe- cific cell population in a patient for the treatment of a disease .
This research line involves (1) technologies used in cell therapy, including direct cell injection systems; bio- reactors and in vitro pre-differentiation; combined drug-cell systems; controlled release systems; noninvasive follow-up and in vivo monitoring systems; (2) analysis of cell biophysical properties (cell channels, membra- ne and cytoskeleton mechanics, etc .), and its response to biophysical stimuli (cellular mechanotransduction, adaptation and plasticity) and (3) modelling the behaviour of the individual cell and of cell populations, down to the organization of tissues and organs .
This line of research has a huge development potential due to the enormous current and future interest of Regenerative Medicine .
BM2. TISSUE ENGINEERING:
• Biomaterials for scaffolds . • Signalling biomolecules . • Cellular and molecular functionalisation of biomaterials . • Mechanobiology and Microfluidics . • Decellularisation and recellularisation of organs and tissues . • Generation of organoids from stem cells: Towards artificial organs .
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