GROUP LEADER - DRUG DELIVERY
My main focus is on pre-clinical translational research. I am especially interested in degenerative disorders of the nervous system and muscle.
In my current role, I am lead scientist in a number of public-funded projects and collaborative programs with pharmaceutical companies, where we explore the efficacy of novel therapeutic targets, and facilitate the in vivo delivery of oligonucleotides and RNA-based therapeutics to improve the biodistribution these drugs.
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
Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1.
Stoodley J. et al, (2023), Int J Mol Sci, 24
A modular RNA delivery system comprising spherical nucleic acids built on endosome-escaping polymeric nanoparticles
Garcia-Guerra A. et al, (2023), Nanoscale Advances
Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy.
Stoodley J. et al, (2023), Methods Mol Biol, 2587, 209 - 237
CELL-PENETRATING PEPTIDE CONJUGATES AND METHODS OF THEIR USE
VARELA MA., (2022)