Nucleic acid therapeutics
Nucleic acid therapeutics are genomic medicines that have the potential to transform human healthcare via the development of novel precision medicines for degenerative diseases, rare diseases and cancers. They therefore have the potential to deliver a paradigm shift in human healthcare. Over the past two decades nucleic acid drugs have demonstrated clear potential to impact human health with landmark approvals for nucleic acid drugs for Duchenne muscular dystrophy and spinal muscular atrophy. Nucleic acid therapeutic drugs operate via diverse mechanisms including via modifying gene expression (single stranded oligonucleotides), silencing genes (double stranded siRNA), switching on genes (mRNA) and editing genes (CRISPR RNA). The Wood laboratory has played a major role in the development of single stranded oligonucleotide drugs for Duchenne muscular dystrophy (Eteplirsen) and other neuromuscular disorders including spinal muscular atrophy and myotonic dystrophy type I. A particular focus of current efforts in the laboratory is on nucleic acid drug delivery. For the great potential nucleic acid drugs to be fully realised the major challenge of effective intracellular delivery of nucleic acid drugs needs to be addressed. A range of nascent technologies now exist with the potential to transform nucleic acid drug delivery including nanotechnologies, lipid based technologies and peptide and protein technologies. Particular efforts in the laboratory are directed towards exosome nanotechnology and peptide/protein technologies with the goal of overcoming the barriers to intracellular nucleic acid drug delivery and understanding the biological requirements for targeted delivery and intelligent control of nucleic acid drugs.