Byun Min Ji, Lim Jaesung, Kim Se-Na, Park Dae-Hwan, Kim Tae-Hyung, Park Wooram, Park Chun Gwon
Department of Biomedical Engineering, SKKU Institute for Convergence, Sungkyunkwan University (SKKU), Suwon, Gyeonggi 16419 Republic of Korea.
Department of Intelligent Precision Healthcare Convergence, SKKU Institute for Convergence, Sungkyunkwan University (SKKU), Suwon, Gyeonggi 16419 Republic of Korea.
Biochip J. 2022;16(2):128-145. doi: 10.1007/s13206-022-00052-5. Epub 2022 Mar 3.
RNA therapeutics, including messenger RNA (mRNA) and small interfering RNA (siRNA), are genetic materials that mediate the translation of genetic direction from genes to induce or inhibit specific protein production. Although the interest in RNA therapeutics is rising globally, the absence of an effective delivery system is an obstacle to the clinical application of RNA therapeutics. Additionally, immunogenicity, short duration of protein expression, unwanted enzymatic degradation, and insufficient cellular uptake could limit the therapeutic efficacy of RNA therapeutics. In this regard, novel platforms based on nanoparticles are crucial for delivering RNAs to the targeted site to increase efficiency without toxicity. In this review, the most recent status of nanoparticles as RNA delivery vectors, with a focus on polymeric nanoparticles, peptide-derived nanoparticles, inorganic nanoparticles, and hybrid nanoparticles, is discussed. These nanoparticular platforms can be utilized for safe and effective RNA delivery to augment therapeutic effects. Ultimately, RNA therapeutics encapsulated in nanoparticle-based carriers will be used to treat many diseases and save lives.
RNA疗法,包括信使核糖核酸(mRNA)和小干扰核糖核酸(siRNA),是介导从基因到诱导或抑制特定蛋白质产生的遗传指令翻译的遗传物质。尽管全球范围内对RNA疗法的兴趣在不断上升,但缺乏有效的递送系统是RNA疗法临床应用的一个障碍。此外,免疫原性、蛋白质表达持续时间短、不必要的酶促降解以及细胞摄取不足可能会限制RNA疗法的治疗效果。在这方面,基于纳米颗粒的新型平台对于将RNA递送至靶位点以提高效率且无毒性至关重要。在本综述中,讨论了纳米颗粒作为RNA递送载体的最新进展,重点是聚合物纳米颗粒、肽衍生纳米颗粒、无机纳米颗粒和杂化纳米颗粒。这些纳米颗粒平台可用于安全有效地递送RNA以增强治疗效果。最终,封装在基于纳米颗粒的载体中的RNA疗法将用于治疗多种疾病并拯救生命。