Abstract
RNA interference (RNAi) is an evolutionarily conserved, endogenous process for post-transcriptional regulation of gene expression. Although RNAi therapeutics have recently progressed through the pipeline toward clinical trials, the application of these as ideal, clinical therapeutics requires the development of safe and effective delivery systems. Inspired by the immense progress with nanotechnology in drug delivery, efforts have been dedicated to the development of nanoparticle-based RNAi delivery systems. For example, a precisely engineered, multifunctional nanocarrier with combined passive and active targeting capabilities may address the delivery challenges for the widespread use of RNAi as a therapy. Therefore, in this review, we introduce the major hurdles in achieving efficient RNAi delivery and discuss the current advances in applying nanotechnology-based delivery systems to overcome the delivery hurdles of RNAi therapeutics. In particular, some representative examples of nanoparticle-based delivery formulations for targeted RNAi therapeutics are highlighted.
Highlights
RNA interference (RNAi) is a cellular mechanism for gene silencing in a sequence-specific manner.RNAi was first observed in plants, later in the worm Caenorhabditis elegans, and subsequently in a wide variety of eukaryotic organisms, including mammals [1,2,3]
The notion that RNAi could lead to a new class of therapeutics caught the attention of many investigators after its discovery, with the launching of clinical trials for approximately twenty small interfering RNAs or short hairpin RNA-based therapeutics for a variety of human diseases [5,6]
The 9-mer Luteinizing Hormone-Releasing Hormones (LHRH) peptide was conjugated to PAMAM dendrimers, whose internal amino group was quaternalized for siRNA loading [90]
Summary
RNA interference (RNAi) is a cellular mechanism for gene silencing in a sequence-specific manner. The notion that RNAi could lead to a new class of therapeutics caught the attention of many investigators after its discovery, with the launching of clinical trials for approximately twenty small interfering RNAs (siRNA, a class of double-stranded RNAs of 20-25 base pairs in length that triggers RNAi) or short hairpin RNA (shRNA)-based therapeutics for a variety of human diseases [5,6]. Such RNAi-based therapeutics include siRNA therapeutics for the treatment of age-related macular degeneration (AMD), diabetic macular edema (DME), and respiratory syncytial virus (RSV) (Table 1).
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