BI 1. Gene therapy and cell 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 concerns 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 specific 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; bioreactors 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, membrane 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 organisation of tissues and organs.
This research line has a huge development potential due to the enormous current and future interest of Regenerative Medicine.
The SCIENTIFIC GOALS of this line are:
BI 2. Tissue engineering:
A paradigm shift is taking place in orthopaedic and reconstructive surgery from using medical devices and tissue grafts to a tissue engineering approach that uses biodegradable scaffolds combined with cells or biological molecules to repair and/or regenerate tissues.
CIBER-BBN studies scaffold-based tissue engineering which includes the development of new materials for scaffolds; the design and use of bioreactors for cell culture; the analysis of the processes involved and the effect of different tissue regeneration stimuli on scaffolds, both in vitro and in vivo; the functionalisation of the scaffold surface; or non invasive follow up and in vitro and in vivo monitoring systems, among many others.
Two new concepts have been added as they are leading research lines in this field: (1) Decellularisation and recellularisation of organs and tissues and (2) generation of organoids from stem cells.
(1) A promising tissue-engineering / regenerative-medicine approach for functional organ replacement has emerged in recent years. Decellularisation of donor organs such as heart, liver, and lung can provide a natural three-dimensional biologic scaffold material that can be seeded with selected cell populations. Preliminary studies in animal models have provided encouraging results for the proof of concept. Some of CIBER-BBN’s research groups are focused on studying it as significant challenges for three-dimensional organ engineering approach still remain.
(2) Generation of transplantable organs using stem cells is a desirable approach for organ replacement and some of CIBER-BBN’s basic and clinical scientist have great interest on it.
The SCIENTIFIC GOALS of this line are:
BI 3. Prostheses and implants
The global objective of this line is to move forward in a new generation of patient-specific prostheses and implants, with greater control over their behaviour and over the evolution of the organ after implantation.
Therefore, this line includes all those elements contributing to the improvement of implant design and features such as: advanced modelling, considering the implant-organ interaction (osseointegration, tissue adaptation, influence of drugs, etc.); systems for supporting surgical decisions; surface mechanisation and functionalisation systems; local and controlled drug release systems operating from the surface of the implant; biomaterials for implantation; intelligent prostheses (active monitoring and control), etc.
The SCIENTIFIC GOALS of this line are:
See spanish website for furher information about research groups participating in this program.
Aída Castellanos
E-Mail: [email protected]
Telephone: (+34) 93 489 44 62 / 679 60 67 44
