Seminar by Prof. Rakesh N. Veedu, Head, Precision Nucleic Acid Theranostics Group, Murdoch University, Australia on "Novel chemically-modified precision nucleic acids for disease specific therapy"

21 Jan 2019
Seminar Room #350
Talk Title : "Novel chemically-modified precision nucleic acids for disease specific therapy"
Abstract
Oligonucleotides - based technologies such as antisense oligonucleotides (AOs), DNAzymes and aptamers attracted significant attention in recent years. We have recently investigated the potential of novel chemica lly - modified AOs containing LNA, HNA, CeNA, modified morpholino s , serinol nucleic acids and other chemistries to modulate RNA splicing in mdx mouse myotubes towards improving the therapy against Duchenne muscular dystrophy. Our results showed that all AOs induced Dmd exon - 23 skipping [1 - 4] . In another study, we investigated the potential of DNAzymes to inhibit integrin alpha - 4 gene transcript ( ITGA - 4 ) towards developing a therapeutic molecule for tackling inflammation in multiple sclerosis. Our results demonstrated that RNV143A, an arm - loop - arm type DNAzyme modified with a 3’ - inverted dT efficiently inhibited ITGA - 4 RNA [5] . Recently, we also de veloped a novel aptamer [6] targeting low - molecular weight amyloid - beta peptides. Our aptamer RNV95 efficiently detected low - molecular weight amyloid beta peptide aggregates in the brain tissue samples of pathologically confirmed Alzheimer disease patients , and offer a great promise towards the diagnosis and therapy of Alzheimer disease [7] . In addition, we also developed novel m uscle cells , and cancer cell s - specific DNA cargo molecules for targeted imaging and the delivery of gene - targeting drugs to muscle s and cancer tissues respectively .