{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Esposito CL"],"funding":["NHLBI NIH HHS","NCI NIH HHS"],"pagination":["1151-1163"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC4048903"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["22(6)"],"pubmed_abstract":["While microRNAs (miRNAs) clearly regulate multiple pathways integral to disease development and progression, the lack of safe and reliable means for specific delivery of miRNAs to target tissues represents a major obstacle to their broad therapeutic application. Our objective was to explore the use of nucleic acid aptamers as carriers for cell-targeted delivery of a miRNA with tumor suppressor function, let-7g. Using an aptamer that binds to and antagonizes the oncogenic receptor tyrosine kinase Axl (GL21.T), here we describe the development of aptamer-miRNA conjugates as multifunctional molecules that inhibit the growth of Axl-expressing tumors. We conjugated the let-7g miRNA to GL21.T and demonstrate selective delivery to target cells, processing by the RNA interference machinery, and silencing of let-7g target genes. Importantly, the multifunctional conjugate reduced tumor growth in a xenograft model of lung adenocarcinoma. Therefore, our data establish aptamer-miRNA conjugates as a novel tool for targeted delivery of miRNAs with therapeutic potential."],"journal":["Molecular therapy : the journal of the American Society of Gene Therapy"],"pubmed_title":["Multifunctional aptamer-miRNA conjugates for targeted cancer therapy."],"pmcid":["PMC4048903"],"funding_grant_id":["R01 CA138503","T32 HL007344","1R01 CA138503"],"pubmed_authors":["Swiderski P","Dassie JP","Santamaria G","Condorelli G","Cerchia L","Giangrande PH","Catuogno S","De Vita G","Esposito CL","de Franciscis V"],"additional_accession":[]},"is_claimable":false,"name":"Multifunctional aptamer-miRNA conjugates for targeted cancer therapy.","description":"While microRNAs (miRNAs) clearly regulate multiple pathways integral to disease development and progression, the lack of safe and reliable means for specific delivery of miRNAs to target tissues represents a major obstacle to their broad therapeutic application. Our objective was to explore the use of nucleic acid aptamers as carriers for cell-targeted delivery of a miRNA with tumor suppressor function, let-7g. Using an aptamer that binds to and antagonizes the oncogenic receptor tyrosine kinase Axl (GL21.T), here we describe the development of aptamer-miRNA conjugates as multifunctional molecules that inhibit the growth of Axl-expressing tumors. We conjugated the let-7g miRNA to GL21.T and demonstrate selective delivery to target cells, processing by the RNA interference machinery, and silencing of let-7g target genes. Importantly, the multifunctional conjugate reduced tumor growth in a xenograft model of lung adenocarcinoma. Therefore, our data establish aptamer-miRNA conjugates as a novel tool for targeted delivery of miRNAs with therapeutic potential.","dates":{"release":"2014-01-01T00:00:00Z","publication":"2014 Jun","modification":"2025-04-18T23:17:22.017Z","creation":"2019-03-27T01:29:40Z"},"accession":"S-EPMC4048903","cross_references":{"pubmed":["24441398"],"doi":["10.1038/mt.2014.5"]}}