Advancements and challenges in mRNA and ribonucleoprotein-based therapies: From delivery systems to clinical applications

IF 6.5 2区医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Molecular Therapy. Nucleic Acids Pub Date : 2024-08-19 DOI:10.1016/j.omtn.2024.102313

Zohre Eftekhari, Horieh Zohrabi, Akbar Oghalaie, Tahereh Ebrahimi, Fatemeh Sadat Shariati, Mahdi Behdani, Fatemeh Kazemi-Lomedasht

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Abstract

The use of mRNA and ribonucleoproteins (RNPs) as therapeutic agents is a promising strategy for treating diseases such as cancer and infectious diseases. This review provides recent advancements and challenges in mRNA- and RNP-based therapies, focusing on delivery systems such as lipid nanoparticles (LNPs), which ensure efficient delivery to target cells. Strategies such as microfluidic devices are employed to prepare LNPs loaded with mRNA and RNPs, demonstrating effective genome editing and protein expression and . These applications extend to cancer treatment and infectious disease management, with promising results in genome editing for cancer therapy using LNPs encapsulating Cas9 mRNA and single-guide RNA. In addition, tissue-specific targeting strategies offer potential for improved therapeutic outcomes and reduced off-target effects. Despite progress, challenges such as optimizing delivery efficiency and targeting remain. Future research should enhance delivery efficiency, explore tissue-specific targeting, investigate combination therapies, and advance clinical translation. In conclusion, mRNA- and RNP-based therapies offer a promising avenue for treating various diseases and have the potential to revolutionize medicine, providing new hope for patients worldwide.

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基于 mRNA 和核糖核蛋白疗法的进展与挑战：从输送系统到临床应用

使用 mRNA 和核糖核蛋白（RNPs）作为治疗剂是治疗癌症和传染病等疾病的一种前景广阔的策略。本综述介绍了基于 mRNA 和 RNP 的疗法的最新进展和挑战，重点是脂质纳米颗粒（LNPs）等递送系统，它们能确保高效递送到靶细胞。利用微流体设备等策略制备装载有 mRNA 和 RNPs 的 LNPs，展示了有效的基因组编辑和蛋白质表达。这些应用已扩展到癌症治疗和传染病管理领域，利用封装 Cas9 mRNA 和单导 RNA 的 LNPs 进行癌症治疗的基因组编辑取得了可喜的成果。此外，组织特异性靶向策略为改善治疗效果和减少脱靶效应提供了潜力。尽管取得了进展，但优化递送效率和靶向性等挑战依然存在。未来的研究应提高递送效率、探索组织特异性靶向、研究联合疗法并推进临床转化。总之，基于 mRNA 和 RNP 的疗法为治疗各种疾病提供了一条前景广阔的途径，并有可能彻底改变医学，为全球患者带来新的希望。

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来源期刊

Molecular Therapy. Nucleic Acids MEDICINE, RESEARCH & EXPERIMENTAL-

CiteScore

15.40

自引率

1.10%

发文量

336

审稿时长

20 weeks

期刊介绍： Molecular Therapy Nucleic Acids is an international, open-access journal that publishes high-quality research in nucleic-acid-based therapeutics to treat and correct genetic and acquired diseases. It is the official journal of the American Society of Gene & Cell Therapy and is built upon the success of Molecular Therapy. The journal focuses on gene- and oligonucleotide-based therapies and publishes peer-reviewed research, reviews, and commentaries. Its impact factor for 2022 is 8.8. The subject areas covered include the development of therapeutics based on nucleic acids and their derivatives, vector development for RNA-based therapeutics delivery, utilization of gene-modifying agents like Zn finger nucleases and triplex-forming oligonucleotides, pre-clinical target validation, safety and efficacy studies, and clinical trials.