Miguel Varela
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.
Recent publications
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Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.
Journal article
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.
Journal article
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
Journal article
Garcia-Guerra A. et al, (2023), Nanoscale Advances
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Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy.
Journal article
Stoodley J. et al, (2023), Methods Mol Biol, 2587, 209 - 237
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CELL-PENETRATING PEPTIDE CONJUGATES AND METHODS OF THEIR USE
Patent
VARELA MA., (2022)