COST Action Members from Spain
Prof. Virginia Arechavala-Gomeza
We work jointly with several international networks of researchers, clinicians, patients and industry that collaborate to find new treatments for neuromuscular disorders.
Our group works in the optimisation of preclinical drug testing in DMD and we have developed new methods to evaluate these therapies using low numbers of cells. Also, we use new CRISPR/Cas gene editing techniques not just as a new potential therapy, but also as a tool to generate better cell culture models to be able to evaluate new treatments more thoroughly before going into clinical trials. We work with human as well as animal cell cultures, so we may collaborate in accelerating the transit to clinical trials as well as minimising the number of animal experiments.
MEMBER OF WG2 & WG4, INTERESTED IN ALL THE WG TASKS
KEYWORDS: Dystrophin quantification, Myoblots, In cell western, Biochemical outcome measures
MODEL: cell culture, human
TISSUE: muscle, skin
DISORDERS: Muscular dystrophies/atrophies, Duchene Muscular Dystrophy, Myotonic dystrophy, Neuromuscular disorders
Dr Maria-Carmen Lopez de las Hazas
MEMBER OF WG1 & WG3
MODEL: humans, mice
TISSUE: Blood or other biofluids, Digestive system, Liver
DISORDERS: Neurological and neurodegenerative diseases, Liver pathology, Metabolic diseases
Prof. Ramon Eritja
Expertise on oligonucleotide and peptide synthesis especially on the methodological aspects of solid-phase synthesis (monomer synthesis, preparation of solid supports, synthesis and purification). Experience in most areas related to the synthesis and properties of oligonucleotide derivatives including non-natural bases, phosphate and backbone modifications, oligonucleotide-peptide conjugates and RNA derivatives. Research efforts have been concentrated in the following areas: 1) Development of novel RNA derivatives to enhance specific gene silencing properties of siRNA by RNA interference, including nuclease resistant siRNAs, lipid-RNA and development of lipid formulation for siRNA transfection. 2) Modulation of the structural parameters of non-canonical DNA structures such as G-quadruplex, i-motif and parallel duplex by chemical modification. 3) Development of oligonucleotides carrying 8-aminopurines for enhanced binding by triple helix formation. 4) Functionalization of DNA bidimensional arrays and DNA origami. 5) Novel linkers for functionalization of nanomaterials. 6) Synthesis and properties of oligonucleotides carrying non-natural nucleobases for the study of mutagenesis, DNA repair, DNA methylase inhibition. 7) Synthesis of oligonucleotide-peptide conjugates.
MEMBER OF WG1, INTERESTED IN WG3 TASKS
KEYWORDS: oligonucleotide synthesis, siRNA, antisense, triplex, quadruplex, i-motif, miRNA
MODEL: cell culture
DISORDERS: Cancer, Colorectal cancer, Liver pathology, Ocular diseases