RNA adopts 3D structures that confer varied functional roles in human biology and
dysfunction in disease. Approaches to therapeutically target RNA structures with small …

Many drugs, or their antecedents, were discovered through observation of their effects on
normal or disease physiology. For the past generation, this phenotypic drug discovery …

RNA-binding proteins (RBPs) are critical effectors of gene expression, and as such their
malfunction underlies the origin of many diseases. RBPs can recognize hundreds of …

Biomedical research is undergoing a paradigm shift towards approaches centred on human
disease models owing to the notoriously high failure rates of the current drug development …

RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …

RNA molecules are essential for cellular information transfer and gene regulation, and
RNAs have been implicated in many human diseases. Messenger and non-coding RNAs …

Alternative splicing (AS) in human genes is widely viewed as a mechanism for enhancing
proteomic diversity. AS can also impact gene expression levels without increasing protein …

Phenotypic drug discovery (PDD) approaches do not rely on knowledge of the identity of a
specific drug target or a hypothesis about its role in disease, in contrast to the target-based …

SMA is an inherited disease that leads to loss of motor function and ambulation and a
reduced life expectancy. We have been working to develop orally administrated …

Recent advances in our understanding of RNA biology have uncovered crucial roles for
RNA in multiple disease states, ranging from viral and bacterial infections to cancer and …