Miguel A. Varela

Miguel is a Senior Research Scientist and Group Leader in the Department of Paediatrics at the University of Oxford, focusing on the development of drug delivery platforms and RNA therapeutics. His research has led to advancements in treating muscle conditions, with peptide-oligonucleotide therapies currently undergoing Clinical Trials. Based at the Institute of Developmental and Regenerative Medicine (IDRM), he also collaborates with industry to fast-track drug discovery efforts.

Publications:    

1. Peptide-conjugated PMOs for the treatment of Myotonic Dystrophy. Stoodley J et al. (2023).  Methods in Molecular Biology
2. Peptide-conjugated Oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice. Klein AF and Varela MA et al. (2019).  Journal of Clinical Investigation
3. Knockdown and replacement therapy mediated by artificial mirtrons in spinocerebellar ataxia 7. Curtis HJ et al. (2017) Nucleic Acids Research
4. Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases. Varela MA et al. (2016). European Journal of Human Genetics.
5. Identification of sequences common to more than one therapeutic target for treating complex diseases: simulating the high variance in sequence interactivity evolved to modulate robust phenotypes. Varela MA et al. (2015). BMC Genomics