Abstract
AbstractThe small interfering RNA (siRNA)‐based therapeutics have raised great attention since the first RNA interference (RNAi)‐derived drug, patisiran, was approved by the US Food and Drug Administration, which represented a landmark in the field of gene therapy. Given the properties of interfering disease‐associated gene expression, RNAi machinery is regarded as an essential factor for preparing precise medicine. However, over the past few years, siRNA drugs are undergoing a period of clinical translation, in which the major hurdle is the limited efficient delivery strategies. Therefore, this mini‐review mainly focuses on describing the state‐of‐the‐art of the nanoscale platforms for delivering siRNA payloads, also addressing their applications in cancer therapy. Finally, the status of siRNA drugs under clinical trials is discussed, providing a comprehensive understanding on the field of oligonucleotide‐mediated therapeutics.
Highlights
In the past few decades, gene therapy has drawn great attention due to the possibility of molecule-manipulation efficacy, for instance, the RNA interference (RNAi)-based therapeutics.[1]
Given that the side effects caused by extracorporeal therapy, such as the disappointed outcomes occurred on adjacent tissues or organs of patients with chemotherapy,[3,4] RNAi therapeutics is prospected to overcome these barriers, thereby reducing disease burden and improving treatment efficiency.[5]
Drug delivery systems based on nanotechnology have made prominent progress, for instance, more than 10 lipid nanoparticle (LNP) systems to carry drugs into targeted tissues or organs have been approved by the Food and Drug Administration (FDA).[7,8]
Summary
In the past few decades, gene therapy has drawn great attention due to the possibility of molecule-manipulation efficacy, for instance, the RNA interference (RNAi)-based therapeutics.[1]. The patisiran (Onpattro) story symbolizes the clinical translation of nanomedicines, which play a significant role on the treatment of multiple diseases.[7] To date, drug delivery systems based on nanotechnology have made prominent progress, for instance, more than 10 lipid nanoparticle (LNP) systems to carry drugs into targeted tissues or organs have been approved by the FDA.[7,8] The natural characteristics of nanoparticles, including low toxicity profiles, wellcontrolled and favorable biocompatibility, endow them great interest for delivering siRNA payloads.[2] Given the challenges that RNAi-based drugs would encounter, using nanoparticles as vectors is likely to reduce these obstacles, followed by enhancing cellular uptake of siRNAs and optimizing silence efficacy.[9] This mini-review aims to briefly describe the fundamental mechanisms of RNAi machinery and provide insights on the current status on the use of nanoparticles for siRNA delivery, as well as to discuss the applications of siRNA in cancer research and clinical therapy
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