RESEARCH TOPIC

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.

POSTER PRESENTED AT INAUGURAL MEETING IN BILBAO

Affiliation

Neuromuscular Disorders,
Virginia Arechavala-Gomeza Lab
Biocruces Bizkaia Health Research Institute

Plaza de Cruces S/N 48903
Barakaldo, Spain

ORCID: 0000-0001-7703-3255

WWW:  www.arechavala-lab.com

ACTION CHAIR

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

RESEARCH TOPIC

Description

Affiliation

Epigenetics of lipid metabolism,
Alberto Davalos Lab
IMDEA Food Institute

Crta Canto Blanco
Madrid, Spain

ORCID: 0000-0001-8199-6724

WWW:  https://www.researchgate.net...

ACTION MEMBER

MEMBER OF WG1 & WG3

KEYWORDS:

MODEL: humans, mice

TISSUE: Blood or other biofluids, Digestive system, Liver

DISORDERS: Neurological and neurodegenerative diseases, Liver pathology, Metabolic diseases

RESEARCH TOPIC

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.

POSTER PRESENTED AT INAUGURAL MEETING IN BILBAO

Affiliation

Institute for advanced chemistry
of catalonia (IQAC),
Nucleic Acids Chemistry Group,
Spanish National Research Council

Jordi Girona 18-26, 08034 Barcelona
Barcelona, Spain

ORCID: 0000-0001-5383-9334

WWW:  https://www.iqac.csic.es/research/departments...

ACTION MEMBER

MEMBER OF WG1, INTERESTED IN WG3 TASKS

KEYWORDS: oligonucleotide synthesis, siRNA, antisense, triplex, quadruplex, i-motif, miRNA

MODEL: cell culture

TISSUE: liver

DISORDERS: Cancer, Colorectal cancer, Liver pathology, Ocular diseases