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儿童肿瘤的核酸治疗

英文原题:Nucleic acid therapy in pediatric cancer.

查看英文原题

Nucleic acid therapy in pediatric cancer.

PubMed 2022/09/09(内容时间) Pharmacol Res Q1 · IF 12.2(JCR 2025)

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中文摘要

过去十年中,微创手术、精准放疗以及多种联合化疗的进步使癌症患者的总体生存期、无进展生存期和生活质量得到改善。此外,免疫检查点抑制剂和免疫细胞疗法等新型治疗手段的发现,为患者和医生抗击癌症提供了助力。而且,在生物相容性和细胞类型靶向纳米载体以及基于核酸的药物用于启动和增强抗肿瘤反应的发展背景下,这一时代已经到来。利用核酸的治疗范式,包括小干扰RNA(siRNA)、反义寡核苷酸(ASO)和信使RNA(mRNA),可以靶向特定蛋白表达以实现治疗效果。已有超过十种核酸治疗药物获得FDA和EMA批准用于罕见病和遗传病,以及数十项针对多种癌症的注册临床试验。尽管过去几十年中观察到儿童癌症通常比成人癌症危险性更低,但儿童癌症在儿童死亡中仍占相当大的比例,给这些家庭带来了极大的痛苦。

因此,有必要更加关注改善儿童癌症的治疗,并发现新的核酸治疗药物,这可能有助于提高治疗效果和预后,从而改善儿童患者的生存期和生活质量。在本综述中,我们聚焦于儿童癌症中的核酸治疗。

展开英文摘要原文

The overall survival, progress free survival, and life quality of cancer patients have improved due to the advance in minimally invasive surgery, precision radiotherapy, and various combined chemotherapy in the last decade.

Furthermore, the discovery of new types of therapeutics, such as immune checkpoint inhibitors and immune cell therapies have facilitated both patients and doctors to fight with cancers.

Moreover, in the context of the development in biocompatible and cell type targeting nano-carriers as well as nucleic acid-based drugs for initiating and enhancing the anti-tumor response have come to the age. The treatment paradigms utilization of nucleic acids, including short interfering RNA (siRNA), antisense oligonucleotides (ASO), and messenger RNA (mRNA), can target specific protein expression to achieve the therapeutic effects.

Over ten nucleic acid therapeutics have been approved by the FDA and EMA in rare diseases and genetic diseases as well as dozens of registered clinical trails for varies cancers. Though generally less dangerous of pediatric cancers than adult cancers was observed during the past decades, yet pediatric cancers accounted for a significant proportion of child deaths which hurt those family very deeply.

Therefore, it is necessary to pay more attention for improving the treatment of pediatric cancer and discovering new nucleic acid therapeutics which may help to improve the therapeutic effect and prognoses in turns to ameliorate the survival period and quality of life for children patient. In this review, we focus on the nucleic acid therapy in pediatric cancers.

论文信息

作者
Li Y、Huang B、Xue Z、Gao Y、Zhuo Z
第一作者单位
Shenzhen Institute for Technology Innovation, National Institute of Metrology, Shenzhen 518055, China; Center for Advanced Measurement Science, National Institute of Metrology, Beijing, China.China
通讯作者单位
Laboratory Animal Center, School of Chemical Biology and Biotechnology, Peking University Shenzhen Graduate School, Shenzhen 518055, China. Electronic address: zhenjianzhuo@163.com.China
文献类型
综述 · 非美国政府资助研究
期刊
Pharmacological research2022 Oct
原文标识
PubMed 36096420 · DOI 10.1016/j.phrs.2022.106441