期刊
ADVANCED DRUG DELIVERY REVIEWS
卷 144, 期 -, 页码 133-147出版社
ELSEVIER
DOI: 10.1016/j.addr.2019.05.004
关键词
siRNA; Nanomaterials; Therapeutics
资金
- Maximizing Investigators' Research Award [R35GM119679]
- National Institute of General Medical Sciences
- College of Pharmacy at The Ohio State University
- Cancer Prevention and Research Institute of Texas (CPRIT) [R1212]
- Welch Foundation [I-1855]
Emerging therapeutics that utilize RNA interference (RNAi) have the potential to treat broad classes of diseases due to their ability to reversibly silence target genes. In August 2018, the FDA approved the first siRNA therapeutic, called ONPATTRO (TM) (Patisiran), for the treatment of transthyretin-mediated amyloidosis. This was an important milestone for the field of siRNA delivery that opens the door for additional siRNA drugs. Currently, >20 small interfering RNA (siRNA)-based therapies are in clinical trials for a wide variety of diseases including cancers, genetic disorders, and viral infections. To maximize therapeutic benefits of siRNA-based drugs, a number of chemical strategies have been applied to address issues associated with efficacy, specificity, and safety. This review focuses on the chemical perspectives behind non-viral siRNA delivery systems, including siRNA synthesis, siRNA conjugates, and nanoparticle delivery using nucleotides, lipids, and polymers. Tracing and understanding the chemical development of strategies to make siRNAs into drugs is important to guide development of additional clinical candidates and enable prolonged success of siRNA therapeutics. (C) 2019 Elsevier B.V. All rights reserved.
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