BackgroundThe immunogenicity and limited stability of conventional messenger RNA (mRNA) has traditionally restricted its potential therapeutic use. In 1992, the first clinical application of mRNA was reported as a potential protein-replacement therapy; however, subsequent investigations have not been made for almost two decades. Recent developments, including increased stability, controlling immunogenicity, as well as utilization of mRNA encoding zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR-Cas9, have implicated modified mRNA as a very promising option for cancer immunotherapy, vaccines, protein expression replacement, and genome editing. This review aims to offer a summary of our present understanding of and improvements in mRNA-based drug technologies, along with a focus on the role in therapeutic options for pediatric respiratory diseases and hemoglobinopathies.ConclusionsThis mini review summarizes the recent advances in modified mRNA-based therapy and its potential therapeutic effect in treating major pediatric diseases.

中文翻译：

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修饰的 mRNA 作为小儿呼吸系统疾病和血红蛋白病的新治疗选择

背景传统信使 RNA (mRNA) 的免疫原性和有限的稳定性传统上限制了其潜在的治疗用途。1992 年，mRNA 的首次临床应用被报道为一种潜在的蛋白质替代疗法；然而，近二十年来一直没有进行后续调查。最近的发展，包括稳定性的提高、免疫原性的控制以及编码锌指核酸酶 (ZFN)、转录激活因子样效应核酸酶 (TALEN) 和 CRISPR-Cas9 的 mRNA 的利用，表明修饰的 mRNA 是一种非常有前途的选择。癌症免疫疗法、疫苗、蛋白质表达替代和基因组编辑。本综述旨在总结我们目前对基于 mRNA 的药物技术的理解和改进，