{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Marban E"],"funding":["NHLBI NIH HHS"],"pagination":["877-885"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11469554"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["135(8)"],"pubmed_abstract":["All Food and Drug Administration-approved noncoding RNA (ncRNA) drugs (n≈20) target known disease-causing molecular pathways by mechanisms such as antisense. In a fortuitous evolution of work on regenerative medicine, my coworkers and I inverted the RNA drug discovery process: first we identified natural disease-modifying ncRNAs, then used them as templates for new synthetic RNA drugs. Mechanism was probed only after bioactivity had been demonstrated. The journey began with the development of cardiosphere-derived cells (CDCs) for cardiac regeneration. While testing CDCs in a first-in-human trial, we discovered they worked indirectly: ncRNAs within CDC-secreted extracellular vesicles mediate the therapeutic benefits. The vast majority of such ncRNAs are fragments of unknown function. We cho"],"journal":["Circulation research"],"pubmed_title":["Deconstructing Regenerative Medicine: From Mechanistic Studies of Cell Therapy to Novel Bioinspired RNA Drugs."],"pmcid":["PMC11469554"],"funding_grant_id":["R01 HL155346","R01 HL167921","R01 HL164588","R01 HL142579","R01 HL168296"],"pubmed_authors":["Marban E"],"additional_accession":[]},"is_claimable":false,"name":"Deconstructing Regenerative Medicine: From Mechanistic Studies of Cell Therapy to Novel Bioinspired RNA Drugs.","description":"All Food and Drug Administration-approved noncoding RNA (ncRNA) drugs (n≈20) target known disease-causing molecular pathways by mechanisms such as antisense. In a fortuitous evolution of work on regenerative medicine, my coworkers and I inverted the RNA drug discovery process: first we identified natural disease-modifying ncRNAs, then used them as templates for new synthetic RNA drugs. Mechanism was probed only after bioactivity had been demonstrated. The journey began with the development of cardiosphere-derived cells (CDCs) for cardiac regeneration. While testing CDCs in a first-in-human trial, we discovered they worked indirectly: ncRNAs within CDC-secreted extracellular vesicles mediate the therapeutic benefits. The vast majority of such ncRNAs are fragments of unknown function. We cho","dates":{"release":"2024-01-01T00:00:00Z","publication":"2024 Sep","modification":"2026-06-03T20:23:40.007Z","creation":"2026-05-01T03:10:14.392Z"},"accession":"S-EPMC11469554","cross_references":{"pubmed":["39325847"],"doi":["10.1161/CIRCRESAHA.124.323058","10.1161/circresaha.124.323058"]}}