Current Transport Systems and Clinical Applications for Small Interfering RNA (siRNA) Drugs
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Current Transport Systems and Clinical Applications for Small Interfering RNA (siRNA) Drugs Fang Liu1 • Chunfang Wang1 • Yuantao Gao2 • Xiao Li1 • Feng Tian1 • Yongtao Zhang1 Mingyang Fu1 • Pengfei Li1 • Yali Wang1 • Fei Wang1
•
Ó Springer International Publishing AG, part of Springer Nature 2018
Abstract Small interfering RNAs (siRNAs) are an attractive new agent with potential as a therapeutic tool because of its ability to inhibit specific genes for many conditions, including viral infections and cancers. However, despite this potential, many challenges remain, including off-target effects, difficulties with delivery, immune responses, and toxicity. Traditional genetic vectors do not guarantee that siRNAs will silence genes in vivo. Rational design strategies, such as chemical modification, viral vectors, and nonviral vectors, including cationic liposomes, polymers, & Chunfang Wang [email protected] Fang Liu [email protected] Yuantao Gao [email protected] Xiao Li [email protected] Feng Tian [email protected] Yongtao Zhang [email protected] Mingyang Fu [email protected] Pengfei Li [email protected] Yali Wang [email protected] Fei Wang [email protected] 1
Shanxi Medical University, No. 56 Xinjian South Road, Yingze District, Taiyuan, China
2
Queen Mary School, Nanchang University, Nanchang, China
nanocarriers, and bioconjugated siRNAs, provide important opportunities to overcome these challenges. We summarize the results of research into vector delivery of siRNAs as a therapeutic agent from their design to clinical trials in ophthalmic diseases, cancers, respiratory diseases, and liver virus infections. Finally, we discuss the current state of siRNA delivery methods and the need for greater understanding of the requirements. Key Points Small interfering RNA (siRNA) drugs have a wide range of applications in the treatment of disease, and a series of siRNA drugs for different diseases have been designed and synthesized. Combined covalent conjugation and carrier envelopes are increasingly being used to overcome problems with siRNA delivery systems. Drugs with different mechanisms can be combined with siRNA drugs to exert synergistic effects of two or more drugs and improve therapeutic effects.
1 Introduction With the rapid development of molecular biotechnology, RNA molecular biology has changed completely since its discovery a little over a decade ago. One of the most important developments is the discovery of ‘‘non-coding RNAs’’ (small RNAs of 20–30 nucleotides that do not encode a protein [2]) that regulate genes and genomes [1]. This regulation can occur at some of the most important levels of genomic function, including chromatin structure,
F. Liu et al.
chromosome segregation, transcription, RNA processing, RNA stability, and translation [1]. Non-coding RNAs have increasingly been found to have more important functions than previously recognized, and many new types of noncoding RNA have been identified. Small RNAs usually inhibit the expression and regulation of genes, so t
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