RNA and Gene Medicine Program

Vision:  To develop new principles for treatment of disease through studies of the basic properties of nucleic acids.

The research focus is on covalent modifications and conformational states of DNA and RNA, and properties of molecules mimicking native nucleic acids.

Cellular regulation of gene expression occurs on many levels including protein-DNA interactions and chromatin structure affecting transcription, and non-coding RNAs (including microRNAs) and posttranscriptional RNA modification affecting translation. The "RNA and Gene Medicine" program focuses on cellular functions of nucleic acids and regulation of gene expression.

Thus we are studying the importance of DNA conformational read-out of the DNA helix for protein-DNA recognition and nucleosome stability and positioning by high resolution chemical probing and bioinformatics approaches Based on our development of a method for profiling of ribose methylations, we are interested in how RNA modifications contribute to ribosome specialization and how changes in modifications promote cancer development. Related to this, we study the structure and function of RNA caps, in particular in relation to mRNA recruitment for translation. RNA interference approaches and sequence specific DNA targeting using peptide nucleic acids (PNA) are exploited for discovery of novel principles for gene therapeutic drugs and their cellular and in vivo delivery with special focus on novel antisense antibiotics and muscular dystrophy. Finally, uranyl photo-technology is used to map protein phosphorylation sites.

Besides the fundamental knowledge in cell function and gene expression obtained in these studies, efforts to convert this knowledge into translational medicine in the areas of cancer,  muscular dystrophy,  infectious and autoimmune diseases, are being pursued.