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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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
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Functional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles.
Journal article
O'Loughlin AJ. et al, (2017), Mol Ther, 25, 1580 - 1587
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C9orf72 and RAB7L1 regulate vesicle trafficking in amyotrophic lateral sclerosis and frontotemporal dementia.
Journal article
Aoki Y. et al, (2017), Brain, 140, 887 - 897
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Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases.
Journal article
Varela MA. et al, (2016), Eur J Hum Genet, 24, 271 - 276