‘RNA therapeutics – more than just for vaccines’ was the theme of one of this year’s Perron Institute Symposia held recently at the University Club of WA. 

Professor Steve Wilton AO, Director of the Perron Institute and the Centre for Molecular Medicine and Innovative Therapeutics (CMMIT) at Murdoch University, provided the opening and closing addresses. 

In collaboration with Professor Sue Fletcher, Professor Wilton has pioneered the development of antisense oligonucleotides (AOs) to overcome the genetic defect underlying Duchenne muscular dystrophy (DMD). Over two decades, Professors Wilton and Fletcher took the idea of using AOs to treat DMD from the laboratory to clinical application via US pharmaceutical company, Sarepta Therapeutics, with three drugs for DMD now approved by the US Food and Drug Administration. 

Ribonucleic acid (RNA) is present in all living cells. It carries out a broad range of functions, from translating genetic information into the molecular structures of the cell to regulating the activity of genes during development and changing environments. Some COVID-19 vaccines are micro or mRNA-based. 

In her talk titled: ‘The mRNA landscape in Australia’, Archa Fox, Associate Professor and ARC Future Fellow in the School of Human Sciences and the School of Molecular Sciences at The University of WA said Australia had been a leader in RNA biology for many years. Cancer and genetic diseases are among the targets for RNA therapeutics, and more recently, the coronavirus, SARS-CoV-2 responsible for COVID-19.   

Professor Merlin Thomas from Melbourne’s Monash University presented on ‘ACE2 alternative splicing and respiratory disease’. ACE2 is a protein connected with SARS-CoV-2 and potentially is a therapeutic target for chronic inflammatory lung conditions. Professor Thomas is a clinician-scientist and program leader in the Department of Diabetes. He is also founder and Chief Scientific Officer of RAGE Biotech and collaborates with Professor Wilton on a project using AO technology in respiratory disorders. 

Dr Tim Yu (PhD and MD), a physician and neurogeneticist at Boston Children’s Hospital and Harvard Medical School, presented his talk from the United States, titledCase studies in patient-customised oligonucleotide medicine’. He spoke about personalised therapies for inherited diseases, including rare disorders, and how he was able to expedite the development of an AO therapy for one young patient so that she could receive targeted treatment just 10 months after diagnosis – a world first.  

Dr Craig McIntosh, a postdoctoral researcher in the Molecular Therapies group at CMMIT, presented on ‘short’ RNAs in rare diseases in his talk titled: ‘The Use of Steric Blocking Antisense Oligonucleotides to Increase Protein Expression’.  

The presentation by Yuval Gurfinkel, a PhD student in the Functional Genomics Research group at CMMIT focused on the gene TDP-43 in sporadic amyotrophic lateral sclerosis (ALS), the most common form of motor neurone disease. His talk was titled: ‘Antisense oligonucleotide suppression of p62-mediated TDP-43 pathology as a treatment for ALS’. 

The gathering was an opportunity to present the Perron Institute’s Three Minute Thesis 2022 and Best Paper Competition Awards. The winners of the Best Paper Competition for the best scientific papers published in 2021 are: 

Early to mid-Career Researcher Best Paper Competition 

First Place Winner – Dr Alex Tang 

Second Place Winner Prize – Dr Stephanie Trend 

Joint Third Place Winner – Dr Chidozie Anyaegbu 

Joint Third Place Winner – Dr Brittney Lins 

PhD Candidate Best Paper Competition 

First Place Winner – Frances Theunissen 

Joint Second Place Prize – Emel Rothzerg 

Joint Second Place Prize – Delin Liu 

Third Place Prize – Jade Kenna 

Special Distinction – Jessica Cale 

Congratulations to the award winners and thank you to the symposium presenters and organisers.