Delivery of oligonucleotide‐based therapeutics: challenges and opportunities
| Autor(a) principal: | |
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| Data de Publicação: | 2021 |
| Outros Autores: | , , , , , , , , , , , , , , , , , , , , |
| Tipo de documento: | Artigo |
| Idioma: | eng |
| Título da fonte: | Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) |
| Texto Completo: | http://hdl.handle.net/10400.18/7983 |
Resumo: | Review |
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Delivery of oligonucleotide‐based therapeutics: challenges and opportunitiesRNA TherapeuticsDeliveryOligonucleotidesAntisense OligonucleotidesPreclinical ModelsSafetyDoenças GenéticasGenetica HumanaReviewNucleic acid-based therapeutics that regulate gene expression have been developed towards clinical use at a steady pace for several decades, but in recent years the field has been accelerating. To date, there are 11 marketed products based on antisense oligonucleotides, aptamers and small interfering RNAs, and many others are in the pipeline for both academia and industry. A major technology trigger for this development has been progress in oligonucleotide chemistry to improve the drug properties and reduce cost of goods, but the main hurdle for the application to a wider range of disorders is delivery to target tissues. The adoption of delivery technologies, such as conjugates or nanoparticles, has been a game changer for many therapeutic indications, but many others are still awaiting their eureka moment. Here, we cover the variety of methods developed to deliver nucleic acid-based therapeutics across biological barriers and the model systems used to test them. We discuss important safety considerations and regulatory requirements for synthetic oligonucleotide chemistries and the hurdles for translating laboratory breakthroughs to the clinic. Recent advances in the delivery of nucleic acid-based therapeutics and in the development of model systems, as well as safety considerations and regulatory requirements for synthetic oligonucleotide chemistries are discussed in this review on oligonucleotide-based therapeutics.This work was supported by funding from Cooperation of Science and Technology (COST) Action CA17103 (networking grant to V.A-G). V.A-G holds a Miguel Servet Fellowship from the ISCIII [grant reference CPII17/00004] that is part-funded by the European Regional Development Fund (ERDF/FEDER) and also acknowledges funding from Ikerbasque (Basque Foundation for Science). S.M.H is funded by the Medical Research Council and Muscular Dystrophy UK. A.A-R receives funding from amongst others the Duchenne Parent Project, Spieren voor Spieren, the Prinses Beatrix Spierfonds, Duchenne UK and through Horizon2020 project BIND. A.G and R.W.J.C are supported by several foundations including the Algemene Nederlandse Vereniging ter Voorkoming van Blindheid, Stichting Blinden-Penning, Landelijke Stichting voor Blinden en Slechtzienden, Stichting Oogfonds Nederland, Stichting Macula Degeneratie Fonds, and Stichting Retina Nederland Fonds (who contributed through UitZicht 2015-31 and 2018-21), together with the Rotterdamse Stichting Blindenbelangen, Stichting Blindenhulp, Stichting tot Verbetering van het Lot der Blinden, Stichting voor Ooglijders, and Stichting Dowilvo; as well as the Foundation Fighting Blindness USA, grant no. PPA-0517-0717-RAD. R.A.M.B is supported by Hersenstichting Nederland Grant DR-2018-00253. G.G. is supported by Ministry of Research and Innovation in Romania/National Program 31N/2016/PN 16.22.02.05. S.A is supported by Project PTDC/BBB-BMD/6301/2014 (Fundação para a Ciência e a Tecnologia—MCTES, Portugal). L.R.D. is supported by Fundación Ramón Areces Grant XVII CN and Spanish Ministry of Science and Innovation (MICINN, grant PID2019-105344RB-I00). T.L is supported by Estonian Research Council grant PSG226. S.K is supported by the Friedrich-Baur-Stiftung. C.F is funded by The Danish Council for Independent Research, Technology and Production Sciences (grant number DFF-4184-00422). W.vRM is supported by ZonMw Programme Translational Research 2 [Project number 446002002], Campaign Team Huntington and AFM Telethon [Project number 20577]. S.E.B is supported by the H2020 projects B-SMART, Grant number 721058, and REFINE, Grant number 761104. A.T.G is supported by the Institut National de la santé et la recherche médicale (INSERM) and the Association Monegasque contre les myopathies (AMM). L.E. is founded by the Association Monegasque contre les myopathies (AMM).EMBO Press/ Wiley Open AccessRepositório Científico do Instituto Nacional de SaúdeHammond, Suzan M.Aartsma‐Rus, AnnemiekeAlves, SandraBorgos, Sven EBuijsen, Ronald A.M.Collin, Rob W.J.Covello, GiuseppinaDenti, Michela A.Desviat, Lourdes R.Echevarría, LucíaFoged, CamillaGaina, GiselaGaranto, AlejandroGoyenvalle, Aurelie T.Guzowska, MagdalenaHolodnuka, IrinaJones, David R.Krause, SabineLehto, TaaviMontolio, MarisolVan Roon‐Mom, WillekeArechavala‐Gomeza, Virginia2022-03-10T16:32:16Z2021-04-062021-04-06T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10400.18/7983engEMBO Mol Med. 2021 Apr 9;13(4):e13243. doi: 10.15252/emmm.202013243. Epub 2021 Apr 6. Review1757-468410.15252/emmm.202013243info:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2023-07-20T15:42:23Zoai:repositorio.insa.pt:10400.18/7983Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-19T18:42:45.966585Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse |
| dc.title.none.fl_str_mv |
Delivery of oligonucleotide‐based therapeutics: challenges and opportunities |
| title |
Delivery of oligonucleotide‐based therapeutics: challenges and opportunities |
| spellingShingle |
Delivery of oligonucleotide‐based therapeutics: challenges and opportunities Hammond, Suzan M. RNA Therapeutics Delivery Oligonucleotides Antisense Oligonucleotides Preclinical Models Safety Doenças Genéticas Genetica Humana |
| title_short |
Delivery of oligonucleotide‐based therapeutics: challenges and opportunities |
| title_full |
Delivery of oligonucleotide‐based therapeutics: challenges and opportunities |
| title_fullStr |
Delivery of oligonucleotide‐based therapeutics: challenges and opportunities |
| title_full_unstemmed |
Delivery of oligonucleotide‐based therapeutics: challenges and opportunities |
| title_sort |
Delivery of oligonucleotide‐based therapeutics: challenges and opportunities |
| author |
Hammond, Suzan M. |
| author_facet |
Hammond, Suzan M. Aartsma‐Rus, Annemieke Alves, Sandra Borgos, Sven E Buijsen, Ronald A.M. Collin, Rob W.J. Covello, Giuseppina Denti, Michela A. Desviat, Lourdes R. Echevarría, Lucía Foged, Camilla Gaina, Gisela Garanto, Alejandro Goyenvalle, Aurelie T. Guzowska, Magdalena Holodnuka, Irina Jones, David R. Krause, Sabine Lehto, Taavi Montolio, Marisol Van Roon‐Mom, Willeke Arechavala‐Gomeza, Virginia |
| author_role |
author |
| author2 |
Aartsma‐Rus, Annemieke Alves, Sandra Borgos, Sven E Buijsen, Ronald A.M. Collin, Rob W.J. Covello, Giuseppina Denti, Michela A. Desviat, Lourdes R. Echevarría, Lucía Foged, Camilla Gaina, Gisela Garanto, Alejandro Goyenvalle, Aurelie T. Guzowska, Magdalena Holodnuka, Irina Jones, David R. Krause, Sabine Lehto, Taavi Montolio, Marisol Van Roon‐Mom, Willeke Arechavala‐Gomeza, Virginia |
| author2_role |
author author author author author author author author author author author author author author author author author author author author author |
| dc.contributor.none.fl_str_mv |
Repositório Científico do Instituto Nacional de Saúde |
| dc.contributor.author.fl_str_mv |
Hammond, Suzan M. Aartsma‐Rus, Annemieke Alves, Sandra Borgos, Sven E Buijsen, Ronald A.M. Collin, Rob W.J. Covello, Giuseppina Denti, Michela A. Desviat, Lourdes R. Echevarría, Lucía Foged, Camilla Gaina, Gisela Garanto, Alejandro Goyenvalle, Aurelie T. Guzowska, Magdalena Holodnuka, Irina Jones, David R. Krause, Sabine Lehto, Taavi Montolio, Marisol Van Roon‐Mom, Willeke Arechavala‐Gomeza, Virginia |
| dc.subject.por.fl_str_mv |
RNA Therapeutics Delivery Oligonucleotides Antisense Oligonucleotides Preclinical Models Safety Doenças Genéticas Genetica Humana |
| topic |
RNA Therapeutics Delivery Oligonucleotides Antisense Oligonucleotides Preclinical Models Safety Doenças Genéticas Genetica Humana |
| description |
Review |
| publishDate |
2021 |
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2021-04-06 2021-04-06T00:00:00Z 2022-03-10T16:32:16Z |
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info:eu-repo/semantics/publishedVersion |
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info:eu-repo/semantics/article |
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article |
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publishedVersion |
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http://hdl.handle.net/10400.18/7983 |
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http://hdl.handle.net/10400.18/7983 |
| dc.language.iso.fl_str_mv |
eng |
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eng |
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EMBO Mol Med. 2021 Apr 9;13(4):e13243. doi: 10.15252/emmm.202013243. Epub 2021 Apr 6. Review 1757-4684 10.15252/emmm.202013243 |
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openAccess |
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EMBO Press/ Wiley Open Access |
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EMBO Press/ Wiley Open Access |
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