Splicing factors are affected by recurrent somatic mutations and copy number variations in
several types of haematologic and solid malignancies, which is often seen as prima facie …

Neurological and neuromuscular diseases resulting from familial, sporadic, or de novo
mutations have devasting personal, familial, and societal impacts. As the initial product of …

A new study combines massively parallel assays, transcriptomics and biophysical modeling
to provide a framework for analyzing the effects of compounds that modulate pre-mRNA …

Motivation RNA is implicated in numerous aberrant cellular functions and disease
progressions, highlighting the crucial importance of RNA-targeted drugs. To accelerate the …

Drug candidates that fail in clinical trials for efficacy reasons might still have favorable safety
and bioavailability characteristics that could be exploited. A failed drug candidate could be …

RNA molecules, similar to proteins, fold into complex structures to confer diverse functions in
cells. The intertwining of functions with RNA structures offers a new therapeutic opportunity …

Splice modulating small molecules have been developed to promote the U1 snRNP to
engage with pre-mRNAs with strong and altered sequence preference. Transcriptomic …