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
Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.
Journal article
Woffindale C. et al, (2026), Mol Ther Nucleic Acids, 37
Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1.
Journal article
González-Martínez I. et al, (2026), Am J Hum Genet
Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for Myotonic Dystrophy type 1
Preprint
González-Martínez I. et al, (2026)
Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy.
Journal article
Moschovaki-Filippidou F. et al, (2025), Brain, 148, 4495 - 4507
miR-107 represses DMPK and is sequestered by CUG repeats triggering the MSI2/miR-7 pathogenesis axis in myotonic dystrophy.
Journal article
Moreno N. et al, (2025), Mol Ther Nucleic Acids, 36