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

Molecular similarity is a key concept in drug discovery. It is based on the assumption that
structurally similar molecules frequently have similar properties. Assessment of similarity …

Expansions of short nucleotide repeats produce several neurological and neuromuscular
disorders including Huntington disease, muscular dystrophy, and amyotrophic lateral …

Oligonucleotides are designed to target RNA using base pairing rules, but they can be
hampered by poor cellular delivery and nonspecific stimulation of the immune system. Small …

The development of small molecules that target RNA is challenging yet, if successful, could
advance the development of chemical probes to study RNA function or precision …

A hypoxic state is critical to the metastatic and invasive characteristics of cancer. Numerous
pathways play critical roles in cancer maintenance, many of which include noncoding RNAs …

Muscleblind-like (MBNL) proteins are key regulators of precursor and mature mRNA
metabolism in mammals. Based on published and novel data, we explore models of tissue …

The recognition of RNA functions beyond canonical protein synthesis has challenged the
central dogma of molecular biology. Indeed, RNA is now known to directly regulate many …

Virtual screening has played a significant role in the discovery of small molecule inhibitors of
therapeutic targets in last two decades. Various ligand and structure-based virtual screening …

Short tandem repeat (STR) or microsatellite, expansions underlie more than 50 hereditary
neurological, neuromuscular and other diseases, including myotonic dystrophy types 1 …