Show simple item record

dc.contributor.authorHammond, Suzan M.
dc.contributor.authorAartsma-Rus, Annemieke
dc.contributor.authorAlves, Sandra
dc.contributor.authorBorgos, Sven Even F.
dc.contributor.authorBuijsen, Ronald A.M
dc.contributor.authorCollin, Rob W.J.
dc.contributor.authorCovello, Giuseppina
dc.contributor.authorDenti, Michela A.
dc.contributor.authorDesviat, Lourdes R.
dc.contributor.authorEchevarria, Lucia
dc.contributor.authorFoged, Camilla
dc.contributor.authorGaina, Gisela
dc.contributor.authorGaranto, Alejandro
dc.contributor.authorGoyenvalle, Aurelie T.
dc.contributor.authorGuzowska, Magdalena
dc.contributor.authorHolodnuka, Irina
dc.contributor.authorJones, David R.
dc.contributor.authorKrause, Sabine
dc.contributor.authorLehto, Taavi
dc.contributor.authorMontolio, Marisol
dc.contributor.authorVan Roon-Mom, Willeke
dc.contributor.authorArechavala-Gomeza, Virginia
dc.description.abstractNucleic 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.en_US
dc.publisherEMBO Pressen_US
dc.rightsNavngivelse 4.0 Internasjonal*
dc.titleDelivery of oligonucleotide‐based therapeutics: challenges and opportunitiesen_US
dc.typePeer revieweden_US
dc.typeJournal articleen_US
dc.rights.holderCopyright: 2021 The Authors. Published under the terms of the CC BY 4.0 licenseen_US
dc.source.journalEMBO Molecular Medicineen_US

Files in this item


This item appears in the following Collection(s)

Show simple item record

Navngivelse 4.0 Internasjonal
Except where otherwise noted, this item's license is described as Navngivelse 4.0 Internasjonal