{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Preußner M"],"funding":["UK Dementia Research Institute","Medical Research Council","Freie Universität Berlin"],"pagination":["e17157"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC10165353"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["15(5)"],"pubmed_abstract":["Neurodegenerative diseases are increasingly prevalent in the aging population, yet no disease-modifying treatments are currently available. Increasing the expression of the cold-shock protein RBM3 through therapeutic hypothermia is remarkably neuroprotective. However, systemic cooling poses a health risk, strongly limiting its clinical application. Selective upregulation of RBM3 at normothermia thus holds immense therapeutic potential. Here we identify a poison exon within the RBM3 gene that is solely responsible for its cold-induced expression. Genetic removal or antisense oligonucleotide (ASO)-mediated manipulation of this exon yields high RBM3 levels independent of cooling. Notably, a single administration of ASO to exclude the poison exon, using FDA-approved chemistry, results in long-"],"journal":["EMBO molecular medicine"],"pubmed_title":["ASO targeting RBM3 temperature-controlled poison exon splicing prevents neurodegeneration in vivo."],"pmcid":["PMC10165353"],"funding_grant_id":["UKDRI‐2001","MRC MC_U132692719"],"pubmed_authors":["Mallucci GR","Scalzitti S","Peretti D","Swinden D","Neumann A","Preußner M","Smith HL","Hughes D","Zhang M","Emmerichs AK","Heyd F","Haltenhof T"],"additional_accession":[]},"is_claimable":false,"name":"ASO targeting RBM3 temperature-controlled poison exon splicing prevents neurodegeneration in vivo.","description":"Neurodegenerative diseases are increasingly prevalent in the aging population, yet no disease-modifying treatments are currently available. Increasing the expression of the cold-shock protein RBM3 through therapeutic hypothermia is remarkably neuroprotective. However, systemic cooling poses a health risk, strongly limiting its clinical application. Selective upregulation of RBM3 at normothermia thus holds immense therapeutic potential. Here we identify a poison exon within the RBM3 gene that is solely responsible for its cold-induced expression. Genetic removal or antisense oligonucleotide (ASO)-mediated manipulation of this exon yields high RBM3 levels independent of cooling. Notably, a single administration of ASO to exclude the poison exon, using FDA-approved chemistry, results in long-","dates":{"release":"2023-01-01T00:00:00Z","publication":"2023 May","modification":"2025-04-25T22:28:05.487Z","creation":"2025-04-06T09:06:17.507Z"},"accession":"S-EPMC10165353","cross_references":{"pubmed":["36946385"],"doi":["10.15252/emmm.202217157"]}}