|
Título del proyecto |
Coordinador |
|
New bone targeted enzyme replacement therapies for pycnodysostosis |
Ibane Abasolo |
|
Improved drug delivery systems for enzyme replacement therapies in Fabry disease. Effect of targeting, pegylation and stabilization of the drug system |
Ibane Abasolo |
|
Increase of the stability, activity and crossing of biological barriers of recombinant proteins through their vehiculization by exosomes and their potential biomedical and industrial use |
Ibane Abasolo |
|
Protein delivery system based on non-covalent PEGylation with telodendrimers |
Miriam Royo |
|
Membrane biophysics in lysosomal storage disorders for improved therapies |
Pau Gorostiza |
|
Introduction, development and support of electrophysiological signal acquisition for the assessment of rare diseases |
Joan F. Alonso López |
|
Hyperspectral and OCT imaging of muscle for assessing muscle degeneration and dystrophia in Duchenne disease |
Olga Conde |
|
Advance characterization of the iron deposits in the frame of mitochondrial diseases |
Lucía Gutiérrez |
|
Development of a non-viral genome-editing platform based on CRISPR/Cas9 and autoassembled lipid nanoparticles to correct genetic mutations in rare diseases |
Gustavo Puras Ochoa |
|
Development of human iPS-derived neural cells as effective drug discovery model for Lafora´s Disease |
Ángel Raya |
|
CRISPR screen of early driver events contributing to glioblastoma development in CMMRD syndrome |
Ángel Raya |
|
Enzyme mutants as tools in the quest for improved therapies for Fabry disease |
Neus Ferrer |
