期刊
EMBO MOLECULAR MEDICINE
卷 13, 期 4, 页码 -出版社
WILEY
DOI: 10.15252/emmm.202013243
关键词
delivery; oligonucleotides; preclinical models; RNA therapeutics; safety
资金
- Cooperation of Science and Technology (COST) Action [CA17103]
- ISCIII - European Regional Development Fund (ERDF/FEDER) [CPII17/00004]
- Ikerbasque (Basque Foundation for Science)
- Medical Research Council
- Muscular Dystrophy UK
- Duchenne Parent Project
- Spieren voor Spieren
- Prinses Beatrix Spierfonds
- Duchenne UK
- Horizon2020 project BIND
- Algemene Nederlandse Vereniging ter Voorkoming van Blindheid
- Stichting Blinden-Penning
- Landelijke Stichting voor Blinden en Slechtzienden
- Stichting Oogfonds Nederland
- Stichting Macula Degeneratie Fonds
- Stichting Retina Nederland Fonds
- Rotterdamse Stichting Blindenbelangen
- Stichting Blindenhulp
- Stichting tot Verbetering van het Lot der Blinden
- Stichting voor Ooglijders
- Stichting Dowilvo
- Foundation Fighting Blindness USA [PPA-0517-0717-RAD]
- Hersenstichting Nederland Grant [DR-2018-00253]
- Ministry of Research and Innovation in Romania/National Program [31N/2016/PN 16.22.02.05]
- Fundacao para a Ciencia e a Tecnologia-MCTES, Portugal [PTDC/BBB-BMD/6301/2014]
- Fundacion Ramon Areces Grant XVII CN
- Spanish Ministry of Science and Innovation (MICINN) [PID2019-105344RB-I00]
- Estonian Research Council grant [PSG226]
- Friedrich-Baur-Stiftung
- Danish Council for Independent Research, Technology and Production Sciences [DFF-4184-00422]
- ZonMw Programme Translational Research 2 [446002002]
- Campaign Team Huntington
- AFM Telethon [20577]
- H2020 project B-SMART [721058]
- H2020 project REFINE [761104]
- Institut National de la sante et la recherche medicale (INSERM)
- Association Monegasque contre les myopathies (AMM)
- Fundação para a Ciência e a Tecnologia [PTDC/BBB-BMD/6301/2014] Funding Source: FCT
The field of nucleic acid-based therapeutics has seen rapid development in recent years, with the main challenge being delivery to target tissues. The adoption of delivery technologies, such as conjugates or nanoparticles, has been a game changer for many therapeutic indications.
Nucleic 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.
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