Splicing is a process necessary for the correct synthesis of proteins and an essential step in gene expression. An impaired minor splicing may lead to aberrant pre-mRNA transcripts and exon skipping, leading to premature stop codons and truncated synthesized proteins, resulting in severe consequences.

Could long non-coding RNAs be the key to developing organ-specific antifibrotic drugs that only mediate their effects in disease-related contexts? That’s the intriguing hypothesis that Haya Therapeutics SA has set out to explore, and its lead program, in heart failure caused by non-obstructive hypertrophic cardiomyopathy, is now in IND-enabling studies. A first clinical trial is pencilled in for late 2024 or early 2025.

A genome-wide look at variants in RNA-binding proteins has revealed that such variants were disproportionately linked to the risk of multiple psychiatric disorders.

Chinese scientists have shown for the first time that the down-regulation of a single RNA-binding protein, polypyrimidine tract-binding protein 1 (Ptbp1), locally converted glial cells to neurons and showed promise for treating the symptoms of neurodegenerative diseases in mice.