Modulation of Pre-mRNA Splicing for Therapeutic Purposes

Date: Monday, 15 January 2024

Time: 11.00AM – 12.00PM

Venue: IMCB Seminar Room 03-46, Level 3 Proteos, Biopolis, Singapore 138673 (Physical)

Speaker: Dr. May Aung-Htut, Murdoch University

Host: Dr. Tommaso Tabaglio, IMCB

 

 

Pre-mRNA splicing is a crucial aspect of gene expression where introns are excised and exons are spliced together. The process is sensitive to changes in our genome as subtle as a single nucleotide and any disturbance in splicing can lead to devastating diseases. Fortunately, splicing can also be modulated, in a controlled manner, using synthetic nucleic acids, known as antisense oligomers, for therapeutic purposes. Our laboratory has developed three antisense oligomers drugs approved by the US Food and Drug Administration as treatments for Duchenne muscular dystrophy. They work by modulating splicing to remove target exons and reframe the dystrophin gene transcript, restoring a semi-functional and truncated dystrophin protein. We are expanding the use of splicing modulating AOs to increase or decrease gene expression or switch which isoforms are expressed, all of which can be applied as therapeutic strategies for both rare and common diseases.

 

Dr Aung-Htut graduated with a PhD in Biochemistry and Molecular Genetics from the University of New South Wales (UNSW) in 2008. Prior to her position as a senior research fellow (Co-Head of Molecular Therapy Laboratory) at the Centre for Molecular Medicine and Innovative Therapeutics (CMMIT), Murdoch University, she held postdoctoral positions at the UNSW and Centre for Cancer biology, University of South Australia working on cellular aging and transcriptional regulation of programmed cell death, respectively. Her current research focuses on designing and evaluating therapeutic antisense oligonucleotides for various diseases, including multiple sclerosis, congenital muscular dystrophy and Pompe’s disease. Her ultimate goal is to extend the application of therapeutic antisense oligonucleotides to other diseases including rare inherited diseases and the leading cause of death, cardiovascular disease. Currently, Dr Aung-Htut and colleagues are driving towards n-of-1 innovative precision therapies for WA children with a rare disease.

 

ALL ARE WELCOME (No registration required)

 

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