Research groups
Miguel A. Varela
GROUP LEADER - DRUG DELIVERY
My research focuses on the molecular mechanisms underlying neuromuscular and cardiac diseases, with the aim of identifying therapeutic targets and developing gene-based therapies.
A key challenge in this field is the effective delivery of nucleic acid therapeutics to target tissues such as skeletal and cardiac muscle. To address this, we have developed a suite of delivery technologies, including peptide and antibody-based systems, some of which are now in clinical translation.
Our work integrates RNA biology, delivery science, and therapeutic development, with the ultimate goal of advancing precision medicines for patients with currently untreatable conditions.
Recent publications
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miR-107 represses DMPK and is sequestered by CUG repeats triggering the MSI2/miR-7 pathogenesis axis in myotonic dystrophy
Moreno N. et al, (2025), Molecular Therapy Nucleic Acids, 36
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TDP-43 regulates LC3ylation in neural tissue through ATG4B cryptic splicing inhibition.
Torres P. et al, (2024), Acta Neuropathol, 148
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Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.
González-Martínez I. et al, (2023), Mol Ther Nucleic Acids, 34
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Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1.
Stoodley J. et al, (2023), Int J Mol Sci, 24
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A modular RNA delivery system comprising spherical nucleic acids built on endosome-escaping polymeric nanoparticles
Garcia-Guerra A. et al, (2023), Nanoscale Advances