Miguel Varela-Muino
Postdoctoral Research Scientist
My focus is on the pre-clinical development of novel oligonucleotide therapeutic drugs for repeat expansion diseases, in particular for Myotonic Dystrophy 1 (DM1).
Recent publications
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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
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A Single Amino Acid Residue Regulates PTEN-Binding and Stability of the Spinal Muscular Atrophy Protein SMN.
Journal article
Rademacher S. et al, (2020), Cells, 9
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Development of LNA Gapmer Oligonucleotide-Based Therapy for ALS/FTD Caused by the C9orf72 Repeat Expansion.
Journal article
Sathyaprakash C. et al, (2020), Methods Mol Biol, 2176, 185 - 208
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Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice.
Journal article
Klein AF. et al, (2019), J Clin Invest
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Knockdown and replacement therapy mediated by artificial mirtrons in spinocerebellar ataxia 7.
Journal article
Curtis HJ. et al, (2017), Nucleic Acids Res, 45, 7870 - 7885