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文章:

肿瘤治疗性反义寡核苷酸:从实验室到临床

Therapeutic Antisense Oligonucleotides in Oncology: From Bench to Bedside

原文发布日期:23 August 2024

DOI: 10.3390/cancers16172940

类型: Article

开放获取: 是

 

英文摘要:

Advancements in our comprehension of tumor biology and chemoresistance have spurred the development of treatments that precisely target specific molecules within the body. Despite the expanding landscape of therapeutic options, there persists a demand for innovative approaches to address unmet clinical needs. RNA therapeutics have emerged as a promising frontier in this realm, offering novel avenues for intervention such as RNA interference and the utilization of antisense oligonucleotides (ASOs). ASOs represent a versatile class of therapeutics capable of selectively targeting messenger RNAs (mRNAs) and silencing disease-associated proteins, thereby disrupting pathogenic processes at the molecular level. Recent advancements in chemical modification and carrier molecule design have significantly enhanced the stability, biodistribution, and intracellular uptake of ASOs, thereby bolstering their therapeutic potential. While ASO therapy holds promise across various disease domains, including oncology, coronary angioplasty, neurological disorders, viral, and parasitic diseases, our review manuscript focuses specifically on the application of ASOs in targeted cancer therapies. Through a comprehensive examination of the latest research findings and clinical developments, we delve into the intricacies of ASO-based approaches to cancer treatment, shedding light on their mechanisms of action, therapeutic efficacy, and prospects.

 

摘要翻译: 

随着对肿瘤生物学及化疗耐药性认识的深入,针对体内特定分子的精准治疗手段得以快速发展。尽管治疗选择日益丰富,临床未满足需求仍亟待创新疗法突破。RNA疗法作为该领域的新兴方向,通过RNA干扰及反义寡核苷酸(ASOs)等干预途径展现出广阔前景。ASOs作为一类多功能治疗剂,能够选择性靶向信使RNA(mRNAs),沉默疾病相关蛋白表达,从而在分子层面阻断病理进程。近年来,化学修饰与载体分子设计的突破显著提升了ASOs的稳定性、生物分布及细胞内摄取效率,进一步增强了其治疗潜力。虽然ASO疗法在肿瘤学、冠状动脉成形术、神经系统疾病、病毒及寄生虫感染等多个疾病领域均具应用前景,本综述将聚焦于ASOs在靶向癌症治疗中的具体应用。通过系统梳理最新研究成果与临床进展,深入探讨基于ASO的癌症治疗策略,阐明其作用机制、疗效特征及发展前景。

 

原文链接:

Therapeutic Antisense Oligonucleotides in Oncology: From Bench to Bedside

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