Miguel Varela
Senior Postdoctoral Scientist / Team Leader
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 PMOs for the Treatment of Myotonic Dystrophy.
Journal article
Stoodley J. et al, (2023), Methods Mol Biol, 2587, 209 - 237
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Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model.
Journal article
Overby SJ. et al, (2022), Mol Ther Nucleic Acids, 27, 1146 - 1155
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Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy.
Journal article
Cerro-Herreros E. et al, (2021), Mol Ther Nucleic Acids, 26, 174 - 191
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Comparable Outcomes of Matched Sibling Donor and Matched Unrelated Donor Stem Cell Transplantation in Children With Acute Leukemia in Argentina.
Journal article
Gómez SM. et al, (2021), J Pediatr Hematol Oncol
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Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1.
Journal article
Marsh S. et al, (2020), Mol Ther, 28, 2527 - 2539