New technologies have greatly shaped the scientific and medical landscape within the
last years. The unprecedented expansion of data and information on RNA biology
has led to the discovery of new RNA classes with unique functions and unexpected
modifications. Today, the biggest challenge is to transfer the large number of findings
in basic RNA biology into corresponding clinical RNA-based therapeutics. Lately, this
research begins to yield positive outcomes. RNA drugs advance to the final phases
of clinical trials or even receive FDA approval. Furthermore, the introduction of the
RNA-guided gene-editing technology CRISPR and advances in the delivery ofmessenger
RNAs have triggered a major progression in the field of RNA-therapeutics. Especially
short interfering RNAs and antisense oligonucleotides are promising examples for novel
categories of therapeutics. However, several issues need to be addressed including
intracellular delivery, toxicity, and immune responses before utilizing RNAs in a clinical
setting. In this review, we provide an overview on opportunities and challenges for clinical
translation of RNA-based therapeutics, with an emphasis on advances in novel delivery
technologies and abdominal aortic aneurysm disease where non-coding RNAs have
been shown to play a crucial regulatory role.
| Identifer | oai:union.ndltd.org:DRESDEN/oai:qucosa:de:qucosa:84541 |
| Date | 04 April 2023 |
| Creators | Schellinger, Isabel N., Dannert, Angelika R., Mattern, Karin, Raaz, Uwe, Tsao, Philip S. |
| Publisher | Frontiers Research Foundation |
| Source Sets | Hochschulschriftenserver (HSSS) der SLUB Dresden |
| Language | English |
| Detected Language | English |
| Type | info:eu-repo/semantics/publishedVersion, doc-type:article, info:eu-repo/semantics/article, doc-type:Text |
| Rights | info:eu-repo/semantics/openAccess |
| Relation | 2297-055X, 571076 |
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