Ruben Artero
Universitat de València (UV), Spain
Therapeutic targeting of MBNL microRNAs as innovative treatments for myotonic dystrophy
Project leader

Ruben Artero
Universitat de València (UV), Spain
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CAIXARESEARCH
AWARDEE -
CONSORTIUM
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Matthew Wood
University of Oxford, UK -
Javier Ramón Azcón
Institute for Bioengineering of Catalonia (IBEC), Spain -
Geneviève Gourdon
Institut IMAGINE, France -
Adolfo López de Munain
Institute Biodonostia, Spain
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HIGHLIGHTS OF
THE PROJECTThe TATAMI project has been instrumental in advancing the development of a new oligonucleotide-based treatment against the rare disease myotonic dystrophy by providing the support required to transition from a basic research result to a biotech fuelled drug program. This fundamentally included addressing the hit-to-lead process in designing antisense oligonucleotides against miR-23b and miR-218, their in vivo validation in two mouse models, and critical confirmation in patient-derived primary myoblasts.
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PROFILE
Dr. Artero is full professor of Genetics at the Universitat de Valencia. He leads the Translational Genomics lab in the UV, which focuses on the molecular basis of muscle atrophy in myotonic dystrophy and in identifying appropriate therapeutic targets and candidate drugs using cell, animal, and bioinformatics approaches. Dr. Artero discovered the MUSCLEBLIND gene, the founding member of the MBNL family of proteins. He has 10 patents and is co-founder of the spin-off company ARTHEx Biotech, which is developing oligonucleotide drugs as therapies for DM1.
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FULL CV
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RESEARCH
INTERESTSHis lab’s research activity is mainly focused on studying mechanisms of pathogenesis and discovering potential pharmacological therapies for rare diseases of genetic origin, in particular myotonic dystrophy (DM1 and DM2), spinal muscular atrophy (SMA), and limb-girdle muscular dystrophy type D2 (LGMDD2). More recently, the lab has become interested in investigating the parallelism between mutation-induced and cancer-induced muscle atrophy (cachexia). For this, the lab uses a comprehensive approach that includes bioinformatics and a whole range of experimental systems, from patient-derived cell cultures and biopsies to animal models like invertebrates (Drosophila) and the mouse. One of the lab’s priority objectives is identifying and repositioning known medicines and developing oligonucleotide drugs against novel therapeutic targets focusing on the translation of research results to society.
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REFERENCES
OR LINKS-
Website of the laboratory:
https://www.uv.es/gt -
More specific about Tatami:
https://arterolab.weebly.com/tatami-project.html -
Includes links to three papers already published reporting results from Tatami, infographics and a short video illustrating the approach we have followed:
https://vimeo.com/604074527
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CONTACT INFO
Department of Genetics, Faculty of Biology, Campus of Burjasot
University of Valencia
Dr. Moliner, 50
46100 Burjasot, Valencia
Spain