期刊
ADVANCED DRUG DELIVERY REVIEWS
卷 178, 期 -, 页码 -出版社
ELSEVIER
DOI: 10.1016/j.addr.2021.113834
关键词
Biomaterials; Therapeutics; Nucleic acid (oligonucleotide) delivery; COVID-19; Vaccines; Cancer; Genetic disease
资金
- National Institutes of Health (NIH) [EB000244]
This review discusses various therapeutic applications of nucleic acids targeted at specific diseases and tissues, highlighting recent technologies that have advanced to late-stage clinical trials and received FDA approval.
Recent medical advances have exploited the ability to address a given disease at the underlying level of transcription and translation. These treatment paradigms utilize nucleic acids - including short interfer-ing RNA (siRNA), microRNA (miRNA), antisense oligonucleotides (ASO), and messenger RNA (mRNA) - to achieve a desired outcome ranging from gene knockdown to induced expression of a selected target pro-tein. Towards this end, numerous strategies for encapsulation or stabilization of various nucleic acid structures have been developed in order to achieve intracellular delivery. In this review, we discuss sev-eral therapeutic applications of nucleic acids directed towards specific diseases and tissues of interest, in particular highlighting recent technologies which have reached late-stage clinical trials and received FDA approval. (c) 2021 Elsevier B.V. All rights reserved.
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