biostudies-literatureUnknown23(4)Weirauch UU1 small nuclear interference (U1i) has recently been described as a novel gene silencing mechanism. U1i employs short oligonucleotides, so-called U1 adaptors, for specific gene knockdown, expanding the field of current silencing strategies that are primarily based on RNA interference (RNAi) or antisense. Despite the potential of U1 adaptors as therapeutic agents, their in vivo application has not yet been studied. Here we explore U1i by analyzing U1 adaptor-mediated silencing of the oncogene Pim-1 in glioblastoma cells. We have generated Pim-1-specific U1 adaptors comprising DNA, locked nucleic acids (LNA), and 2'-O-Methyl RNA and demonstrate their ability to induce a Pim-1 knockdown, leading to antiproliferative and pro-apoptotic effects. For the therapeutic in vivo application of U1 adaNucleic acid therapeutics264-72https://www.ebi.ac.uk/biostudies/studies/S-EPMC3723237biostudies-literatureU1 adaptors for the therapeutic knockdown of the oncogene pim-1 kinase in glioblastoma.PMC3723237Grunweller ACuellar LAigner AWeirauch UHartmann RKfalseU1 adaptors for the therapeutic knockdown of the oncogene pim-1 kinase in glioblastoma.U1 small nuclear interference (U1i) has recently been described as a novel gene silencing mechanism. U1i employs short oligonucleotides, so-called U1 adaptors, for specific gene knockdown, expanding the field of current silencing strategies that are primarily based on RNA interference (RNAi) or antisense. Despite the potential of U1 adaptors as therapeutic agents, their in vivo application has not yet been studied. Here we explore U1i by analyzing U1 adaptor-mediated silencing of the oncogene Pim-1 in glioblastoma cells. We have generated Pim-1-specific U1 adaptors comprising DNA, locked nucleic acids (LNA), and 2'-O-Methyl RNA and demonstrate their ability to induce a Pim-1 knockdown, leading to antiproliferative and pro-apoptotic effects. For the therapeutic in vivo application of U1 ada2013-01-01T00:00:00Z2013 Aug2025-04-04T08:01:03.857Z2019-03-27T01:13:37ZS-EPMC37232372372478010.1089/nat.2012.0407