Nucleic acid therapeutics
The use of DNA, RNA and related analogues as drugs could revolutionise many disease areas of therapy, including cancers, immune disorders, bacterial and viral infections.
One of the key barriers to application of these exciting new therapies is the formulation of DNA and RNA, which are easily degradable hydrophilic biomolecules. We are investigating new materials and formulation chemistries which will facilitate protection and transport of these nucleic acids to the required cellular compartment in disease sites in the body, and biophysical and biological methods to characterise these formulations. We are also interested in their transport and the downstream effects of the nucleic acids in cell lines and in vivo.
Complementary work is also taking place within Molecular Therapeutics and Formulation (MTF) looking at the fundamental biology of DNA, RNA and its transcription, translation and expression to elucidate how we can exploit these nucleic acids more effectively as treatments in various diseases. Projects in this area are therefore spread throughout other themes within MTF particularly Biopharmaceuticals, Nanomaterials Formulation and Characterisation, and RNA in Health and Disease, as well as collaborations with researchers in the Division of Advanced Materials and Heathcare Technologies.
Microscopy images show delivery of RNA (red) in A549-luciferase expressing cells by polymer carriers. Cell nuclei stained with DAPI (blue) a) untreated cells, b) naked siRNA, c-e) three different polymer RNA formulations, with highest RNA delivery shown in (e) Scale bar: 20 m.
TEM of RNA formulation showing uniform size (30-40nm), tightly condensed RNA core and surface PEG layer for stabilisation.