Ministerio de Ciencia e Innovación

Enfermedades Raras

Título del proyecto


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

Otras colaboraciones intramurales