New treatments for rare diseases
The Molecular Therapy Laboratory is led by Professor Steve Wilton AO and Dr May Aung-Htut and is located at the Personalised Medicine Centre at Murdoch University (a joint venture with the Perron Institute).
In 2016, the USA Food and Drug Administration (FDA) gave accelerated approval to a new treatment for Duchenne Muscular Dystrophy (DMD) using drugs created by Professors Steve Wilton and Sue Fletcher and the Molecular Therapy Laboratory. Eteplirsen (Exondys 51) is the first dystrophin restoring drug of its type ever approved by the FDA. In 2019, the FDA granted accelerated approval for Golodirsen (Vyondys 53), targeting another subset of DMD, and in 2021 a third approval was granted for Casimersen (Amondys 45). These three drugs can treat almost thirty per cent of patients diagnosed with DMD.
The focus of our Molecular Therapy Laboratory is the use of small genetic ‘patches’ called antisense oligonucleotides (ASOs) to mask part of a genetic message associated with a particular inherited disease. In the case of DMD, the defective genetic message is associated with the gene for the protein dystrophin, which plays a pivotal role in maintaining muscle structure and integrity.
Make a submission for antisense drug development
Thanks to generous support from the Stan Perron Charitable Foundation, the Perron Institute for Neurological and Translational Science, and Neuromuscular WA, we are working to improve genetic diagnosis and develop potential treatments for certain conditions.
If you are a clinician in seeking molecular diagnosis and development of suitable antisense oligomer drugs for amenable conditions, complete the form below. We'll review your submission and be in contact within a week.
Make a submission
Our research team
Professor Steve Wilton AO
Co-head
Professor Steve Wilton AO
Co-head
Professor Steve Wilton is the Director of the Perron Institute, Foundation Chair of Molecular Therapy at Murdoch University and Deputy Director of Murdoch University’s Personalised Medicine Centre. With Professor Sue Fletcher, Professor Wilton pioneered the use of antisense oligonucleotides (AOs) to overcome dystrophin gene defects causing Duchenne muscular dystrophy (DMD). Professor Wilton has received multiple awards for his research, including the Eureka Prize for Medical Research Translation, the Western Australian Innovator of the Year award and Officer of the Order of Australia.
Academic profile
Dr May Aung-Htut is the Co-Head of the Molecular Therapy Laboratory. A PhD graduate from the University of New South Wales, May previously held postdoctoral positions at the UNSW and University of South Australia working on cellular aging and transcriptional regulation of programmed cell death. Her current research focuses on therapeutic antisense oligonucleotides for various diseases, including multiple sclerosis, congenital muscular dystrophy and Pompe’s disease. Her ultimate goal is to extend this research to other diseases, including rare inherited diseases and cardiovascular disease.
Academic profile
Dr Jessica Cale
Research fellow
Dr Jessica Cale
Research fellow
Dr Jessica Cale is a Research Fellow with the Molecular Therapy Laboratory. She completed her PhD at Murdoch in 2021 under the supervision of Professor Steve Wilton AO and Professor Sue Fletcher AO, specialising in antisense therapies for Malan syndrome, a rare connective tissue disorder. Jessica has been involved in commercial-in-confidence, industry-funded research contracts with Senisca Ltd. (UK) and Lyramid Ltd. (Australia), where her work on antisense oligonucleotide therapies for healthy aging and cancer resulted in being named as an inventor on an international patent application.
Academic profile
Dr Kelly Martinovich
Research Fellow
Dr Kelly Martinovich
Research Fellow
Dr Kelly Martinovich is a Research Fellow at the Molecular Therapy Laboratory. Kelly completed her PhD in 2021 at the University of Western Australia investigating oligo based therapeutics for people with rare Cystic Fibrosis variants. As a highly skilled Lab-based researcher, she has advanced the understanding of many cell and molecular biology methodologies, contributing to novel discoveries in Airway Diseases, Infectious Diseases, Cardiovascular Diseases and oligo-based therapeutics. She is focused on developing novel therapeutics and bridging the gap between academia and industry.
Academic profile
Dr Karina Yui Eto
Postdoctoral Scientist
Dr Karina Yui Eto
Postdoctoral Scientist
Dr Karina Yui Eto is a postdoctoral scientist with the Molecular Therapy Laboratory. Karina obtained her PhD from the University of Western Australia under the supervision of Professor Charlie Bond and Associate Professor Josh Ramsay, where she specialised in protein-nucleic acids interactions. Karina’s research has focused on the development of antisense oligonucleotide-mediated treatment strategies for individual with rare disorders. Karina was part of the Motor Neurone Disease Genetics and Therapeutics Research group at the Perron Institute and Functional Genomics Research group.
Academic profile
Dr Vidya Krishnan
Postdoctoral Scientist
Dr Vidya Krishnan
Postdoctoral Scientist
Dr Vidya Krishnan is currently working as a postdoctoral researcher with the Molecular Therapy Laboratory. Vidya has a first-class Masters in Biotechnology and completed her PhD in Neuroscience in 2016 at the University of Western Australia. Her work is focussed on evaluating the efficacy of isoform-switching antisense oligonucleotides to prevent or delay muscle damage via inflammation in animal models of muscular dystrophies. She is also working on assessing the biodistribution of different antisense oligonucleotides chemistries in murine models.
Academic profile
Dr Anuradha Sooda
Postdoctoral Scientist
Dr Anuradha Sooda
Postdoctoral Scientist
Dr. Anuradha Sooda is a postdoctoral research scientist in the Molecular Therapy Laboratory led by Dr. May Aung-Htut and Professor Steve Wilton. Anu earned her PhD from Murdoch University in 2020, after completing an MSc at Massey University, New Zealand. With over 10 years of research expertise in life sciences, she specialises in cell biology, molecular biology, immunology, and bioinformatics. Anu’s current research focuses on applying bioinformatics and machine learning to molecular therapy research, advancing our understanding of genetic associations and therapeutic interventions.
Academic profile
Senior Research Officers
- Abbie Adams
- Russell Johnsen
- Kane Greer
- Robert Smith
Students
- Isabella Trew (PhD)
- Aidan Murphy (PhD)
- Sophie Chapman (Honours)
- Krishna Karthik Gabbita (Masters)
Research Assistants
- Dr Penny Nice
- Will Johnsen
- Erin Bolitho
- Giselle Sugianto
- Caitlyn Vicars
Research Officers
- Kristin Ham (also PhD student)
Contact us
If you have any questions, please contact us at moleculartherapy@murdoch.edu.au.
