{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Connerty P"],"funding":["German Research Foundation","National Health and Medical Research Council","Tour De Cure, Australia","Cancer Council Australia","Australian Research Council"],"pagination":["1681"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC8539450"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["13(10)"],"pubmed_abstract":["Standard of care therapies for children with acute myeloid leukemia (AML) cause potent off-target toxicity to healthy cells, highlighting the need to develop new therapeutic approaches that are safe and specific for leukemia cells. Long non-coding RNAs (lncRNAs) are an emerging and highly attractive therapeutic target in the treatment of cancer due to their oncogenic functions and selective expression in cancer cells. However, lncRNAs have historically been considered 'undruggable' targets because they do not encode for a protein product. Here, we describe the development of a new siRNA-loaded lipid nanoparticle for the therapeutic silencing of the novel oncogenic lncRNA LINC01257. Transcriptomic analysis of children with AML identified LINC01257 as specifically expressed in t(8;21) AML an"],"journal":["Pharmaceutics"],"pubmed_title":["Development of siRNA-Loaded Lipid Nanoparticles Targeting Long Non-Coding RNA LINC01257 as a Novel and Safe Therapeutic Approach for t(8;21) Pediatric Acute Myeloid Leukemia."],"pmcid":["PMC8539450"],"funding_grant_id":["PG16-01","FR 854/8-1","APP1157871","APP1119152","CE140100036","Grant #: RSP-00122-19/20","APP1059804","APP1091261"],"pubmed_authors":["Kavallaris M","de Bock CE","Mayoh C","Lock RB","Moles E","Connerty P","Smith JL","Meshinchi S","Jayatilleke N"],"additional_accession":[]},"is_claimable":false,"name":"Development of siRNA-Loaded Lipid Nanoparticles Targeting Long Non-Coding RNA LINC01257 as a Novel and Safe Therapeutic Approach for t(8;21) Pediatric Acute Myeloid Leukemia.","description":"Standard of care therapies for children with acute myeloid leukemia (AML) cause potent off-target toxicity to healthy cells, highlighting the need to develop new therapeutic approaches that are safe and specific for leukemia cells. Long non-coding RNAs (lncRNAs) are an emerging and highly attractive therapeutic target in the treatment of cancer due to their oncogenic functions and selective expression in cancer cells. However, lncRNAs have historically been considered 'undruggable' targets because they do not encode for a protein product. Here, we describe the development of a new siRNA-loaded lipid nanoparticle for the therapeutic silencing of the novel oncogenic lncRNA LINC01257. Transcriptomic analysis of children with AML identified LINC01257 as specifically expressed in t(8;21) AML an","dates":{"release":"2021-01-01T00:00:00Z","publication":"2021 Oct","modification":"2025-08-16T03:05:44.21Z","creation":"2024-11-09T18:37:45.269Z"},"accession":"S-EPMC8539450","cross_references":{"pubmed":["34683974"],"doi":["10.3390/pharmaceutics13101681"]}}