Design and development of antisense RNA therapies for neurometabolic diseases
Pathophysiology and Therapy of Genetic Diseases
Centro de Biología Molecular “Severo Ochoa” CSIC-UAM (CBMSO)
DESCRIPTION OF THE OFFER
The research group (www.cbm.uam.es/lab220 ) works in the in vitro and in vivo use of antisense therapy to revert splicing defects in different rare neurometabolic diseases, participating in a EU-COST Actions within this topic. We are using gene editing CRISPR/Cas9 technology for the generation of cellular and animal models with specific splicing mutations, to identify and test candidate therapeutical antisense oligonucleotides. The project will cover different aspects within this topic, from the design and testing of antisense oligonucleotides in cell lines (bioinformatics analysis, cell culture, transfections, RT-PCR, Western blot analysis…) to the characterization of newly generated using CRISPR/Cas cellular and mouse models with patient-specific splicing mutations (biochemical and cellular phenotyping).
We are looking for motivated students who would like to pursue a scientific career doing a PhD after the Master.
Biomolecules & Cell D.