Research groups
Miguel A. Varela
GROUP LEADER - DRUG DELIVERY
My research sits at the intersection of RNA biology, nucleic acid chemistry, and therapeutic development, with a focus on designing and delivering antisense-based medicines for conditions that currently lack effective treatments.
A key focus of my group is the delivery of nucleic acid therapeutics to tissues that remain difficult to reach, developing peptide- and antibody-conjugated systems that improve tissue uptake and selectivity. Some of this delivery work is progressing towards clinical translation.
Our work integrates chemistry, delivery science, and translational strategy, with the goal of building a platform that can be rapidly adapted across disease indications to deliver precision medicines to patients.
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, 113, 529 - 547
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