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Drug-inducible systems allowing the control of transgene expression and knockdown in mammalian cells are invaluable tools for genetic research, and could also play important roles in translational research or gene therapy. We and others have developed a le ...
The delivery of molecules and genes to the central nervous system (CNS) poses a major challenge for the treatment of neurodegenerative diseases. CNS disorders require long-term intervention and the presence of the blood-brain barrier (BBB) restricts the pe ...
Recombinant protein-based biologics are increasingly relevant in the pharmaceutical industry. Currently, mammalian cell-based bioprocesses are a routine manufacturing procedure for complex proteins such as recombinant antibodies, and the number of new biol ...
Retrograde transport of viral vectors in the rodent spinal cord provides a powerful means to administer a therapeutic transgene from the innervated musculature. With the aim of scaling up this approach to non-human primates, we have injected recombinant ad ...
Insertional mutagenesis represents a serious adverse effect of gene therapy with integrating vectors. However, although uncontrolled activation of growth-promoting genes in stem cells can predictably lead to oncological processes, this is far less likely i ...
The delivery of exogenous nucleic acids into mammalian cells is a valuable technique for basic research studies on the expression, regulation and function of genes and proteins. At the same time, gene delivery to cultivated mammalian cells has become funda ...
Tissue Engineering points to maintain, restore or to improve and create new tissues. This purpose typically involves the selection of a natural or synthetic biomaterial as a scaffold to create the biological substitutes. Furthermore, angiogenesis, the grow ...
Animal models of human pathologies remain invaluable tools for unraveling disease mechanisms and evaluating potential therapeutic strategies. For a number of diseases, the lack of a reliable animal model represents an important limiting step towards the de ...
Throughout these last years, many synthetic polymers have been developed for non-viral gene delivery systems in attempt to overcome potential shortcomings of viral vectors. However, it is still difficult to achieve highly efficient transfection under physi ...
Haematopoeitic system diseases, both acquired and inherited, can be currently cured by allogeneic haematopoietic stem cell transplantation. This treatment provides highly successful immune function recovery for patients receiving grafts of HLA-compatible d ...