RBM3-inducing antisense oligonucleotide

RBM3-inducing antisense oligonucleotide is a splice-switching antisense oligonucleotide (ASO) designed to selectively upregulate the expression of the cold-shock protein RNA-binding motif 3 (RBM3) under normothermic conditions. Developed by researchers at the Freie Universität Berlin, the University of Cambridge, and the UK Dementia Research Institute, the ASO targets and blocks a temperature-controlled "poison exon" (E3a) within the RBM3 pre-mRNA. Under normal warm temperatures, this poison exon is included, leading to nonsense-mediated decay (NMD) of the transcript. By sterically blocking the splice-regulatory sequences of E3a, the ASO promotes its exclusion, thereby increasing canonical RBM3 mRNA and protein levels. This "hypothermia in a syringe" approach aims to harness the neuroprotective, anti-apoptotic, and synapse-regenerating effects of therapeutic hypothermia without the systemic risks of cooling. It is being investigated preclinically for neurodegenerative diseases (such as prion disease, Alzheimer's disease, and Parkinson's disease) and acute neurological conditions like temporal lobe epilepsy and status epilepticus.

Other names

ASO against E3aRBM3-inducing ASORBM-3-inducing ASORBM 3-inducing ASO