RNA-based therapy in the management of lipid disorders: a review

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dc.contributor.authorBlom, Dirk J
dc.contributor.authorMarais, Adrian D
dc.contributor.authorMoodley, Rajen
dc.contributor.authorvan der Merwe, Nico
dc.contributor.authorvan Tonder, Alet
dc.contributor.authorRaal, Frederick J
dc.date.accessioned2022-04-26T08:07:47Z
dc.date.available2022-04-26T08:07:47Z
dc.date.issued2022-04-23
dc.date.updated2022-04-24T03:23:59Z
dc.description.abstractThis review focuses on antisense oligonucleotides and small interfering ribonucleic acid therapies approved or under development for the management of lipid disorders. Recent advances in RNA-based therapeutics allow tissue-specific targeting improving safety. Multiple potential target proteins have been identified and RNA-based therapeutics have the potential to significantly improve outcomes for patients with or at risk for atherosclerotic cardiovascular disease. The advantages of RNA-based lipid modifying therapies include the ability to reduce the concentration of almost any target protein highly selectively, allowing for more precise control of metabolic pathways than can often be achieved with small molecule-based drugs. RNA-based lipid modifying therapies also make it possible to reduce the expression of target proteins for which there are no small molecule inhibitors. RNA-based therapies can also reduce pill burden as their administration schedule typically varies from weekly to twice yearly injections. The safety profile of most current RNA-based lipid therapies is acceptable but adverse events associated with various therapies targeting lipid pathways have included injection site reactions, inflammatory reactions, hepatic steatosis and thrombocytopenia. While the body of evidence for these therapies is expanding, clinical experience with these therapies is currently limited in duration and the results of long-term studies are eagerly awaited.en_US
dc.identifier.apacitationBlom, D. J., Marais, A. D., Moodley, R., van der Merwe, N., van Tonder, A., & Raal, F. J. (2022). RNA-based therapy in the management of lipid disorders: a review. <i>Lipids in Health and Disease</i>, 21(1), 41. http://hdl.handle.net/11427/36402en_ZA
dc.identifier.chicagocitationBlom, Dirk J, Adrian D Marais, Rajen Moodley, Nico van der Merwe, Alet van Tonder, and Frederick J Raal "RNA-based therapy in the management of lipid disorders: a review." <i>Lipids in Health and Disease</i> 21, 1. (2022): 41. http://hdl.handle.net/11427/36402en_ZA
dc.identifier.citationBlom, D.J., Marais, A.D., Moodley, R., van der Merwe, N., van Tonder, A. & Raal, F.J. 2022. RNA-based therapy in the management of lipid disorders: a review. <i>Lipids in Health and Disease.</i> 21(1):41. http://hdl.handle.net/11427/36402en_ZA
dc.identifier.ris TY - Journal Article AU - Blom, Dirk J AU - Marais, Adrian D AU - Moodley, Rajen AU - van der Merwe, Nico AU - van Tonder, Alet AU - Raal, Frederick J AB - This review focuses on antisense oligonucleotides and small interfering ribonucleic acid therapies approved or under development for the management of lipid disorders. Recent advances in RNA-based therapeutics allow tissue-specific targeting improving safety. Multiple potential target proteins have been identified and RNA-based therapeutics have the potential to significantly improve outcomes for patients with or at risk for atherosclerotic cardiovascular disease. The advantages of RNA-based lipid modifying therapies include the ability to reduce the concentration of almost any target protein highly selectively, allowing for more precise control of metabolic pathways than can often be achieved with small molecule-based drugs. RNA-based lipid modifying therapies also make it possible to reduce the expression of target proteins for which there are no small molecule inhibitors. RNA-based therapies can also reduce pill burden as their administration schedule typically varies from weekly to twice yearly injections. The safety profile of most current RNA-based lipid therapies is acceptable but adverse events associated with various therapies targeting lipid pathways have included injection site reactions, inflammatory reactions, hepatic steatosis and thrombocytopenia. While the body of evidence for these therapies is expanding, clinical experience with these therapies is currently limited in duration and the results of long-term studies are eagerly awaited. DA - 2022-04-23 DB - OpenUCT DP - University of Cape Town IS - 1 J1 - Lipids in Health and Disease KW - RNA-based therapies KW - Lipid disorders KW - Small interfering RNA KW - Antisense oligonucleotides KW - Lipid-lowering KW - LDL-C LK - https://open.uct.ac.za PY - 2022 T1 - RNA-based therapy in the management of lipid disorders: a review TI - RNA-based therapy in the management of lipid disorders: a review UR - http://hdl.handle.net/11427/36402 ER - en_ZA
dc.identifier.urihttps://doi.org/10.1186/s12944-022-01649-3
dc.identifier.urihttp://hdl.handle.net/11427/36402
dc.identifier.vancouvercitationBlom DJ, Marais AD, Moodley R, van der Merwe N, van Tonder A, Raal FJ. RNA-based therapy in the management of lipid disorders: a review. Lipids in Health and Disease. 2022;21(1):41. http://hdl.handle.net/11427/36402.en_ZA
dc.language.isoenen_US
dc.language.rfc3066en
dc.publisher.departmentDivision of Lipidologyen_US
dc.publisher.facultyFaculty of Health Sciencesen_US
dc.rights.holderThe Author(s)
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/en_US
dc.sourceLipids in Health and Diseaseen_US
dc.source.journalissue1en_US
dc.source.journalvolume21en_US
dc.source.pagination41en_US
dc.source.urihttps://lipidworld.biomedcentral.com/
dc.subjectRNA-based therapiesen_US
dc.subjectLipid disordersen_US
dc.subjectSmall interfering RNAen_US
dc.subjectAntisense oligonucleotidesen_US
dc.subjectLipid-loweringen_US
dc.subjectLDL-Cen_US
dc.titleRNA-based therapy in the management of lipid disorders: a reviewen_US
dc.typeJournal Articleen_US
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