Epigenetic Modifiers and Synthetic Lethality in Cancer Therapy: Emerging Targets Beyond Traditional Approaches.

IF 3.6 4区 医学 Q3 CHEMISTRY, MEDICINAL
Venkatesh Kamath, Vasudev Pai, Bhavana Bhat, Chandrasekhar K S, Pawan G Nayak, Aravinda Pai
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Abstract

As the field of anticancer drug development is constantly changing, combining synthetic lethality with epigenetic modifiers opens up new possibilities for targets that fall outside the traditional drug target range. Enzymes involved in DNA methylation and histone modification are examples of epigenetic modifiers that promote gene expression without altering the DNA sequence of the gene. These mechanisms play a key role in carcinogenesis when they are altered, as they downregulate tumor suppressor genes and overexpress oncogenic pathways. Synthetic lethality is a phenomenon in which simultaneous mutations or perturbations of two genes result in cell death, but alterations to one gene alone do not cause cell death. It was first observed in genetic research conducted on model organisms, such as fruit flies and fungi. The most well-known example of this idea from the perspective of cancer treatment is PARP inhibitors, which are effective in tumors with BRCA1/2 mutations, where further failure of DNA repair results in cell sensitization. Building on the concept of synthetic lethality, current research focuses on exploiting epigenetic flaws that are common in cancer cells. For example, when chromatin remodelers or methyltransferases cease to function, malignant cells undergo genetic rewiring, rendering them vulnerable to treatment. Recent research has produced some striking examples of synthetic-lethal drug interactions and biomarkers used in metagenomics for personalized medicine by targeting the secondary pathways used by cancer cells as a result of primary loss-of-function mutations, which selectively kill cancer cells while sparing healthy cells. The discovery of actionable epigenetic dependencies and overcoming tumor heterogeneity remain the largest challenges in translating these fascinating scientific discoveries to the clinic. The convergence of epigenetic modulators with synthetic-lethality-based therapeutic architectures is poised to define a transformative paradigm in precision oncology. By orchestrating multilayered perturbations across chromatin-regulatory networks, DNA damage-response pathways, and context-specific vulnerability nodes, this integrative strategy surpasses the limitations of conventional target-centric pharmacology and enables mechanistically rational, synergistic antitumor interventions.

癌症治疗中的表观遗传修饰剂和合成致死率:超越传统方法的新兴靶点。
随着抗癌药物开发领域的不断变化,将合成致死性与表观遗传修饰剂相结合,为传统药物靶点范围之外的靶点开辟了新的可能性。参与DNA甲基化和组蛋白修饰的酶是表观遗传修饰剂的例子,它们促进基因表达而不改变基因的DNA序列。当这些机制发生改变时,它们下调肿瘤抑制基因并过度表达致癌途径,从而在致癌过程中发挥关键作用。合成致死是一种现象,其中两个基因同时突变或扰动导致细胞死亡,但单独改变一个基因不会导致细胞死亡。这是在果蝇和真菌等模式生物的遗传研究中首次观察到的。从癌症治疗的角度来看,这一观点最著名的例子是PARP抑制剂,它对BRCA1/2突变的肿瘤有效,其中DNA修复的进一步失败导致细胞致敏。基于合成致死率的概念,目前的研究重点是利用癌细胞中常见的表观遗传缺陷。例如,当染色质重塑酶或甲基转移酶停止发挥作用时,恶性细胞会经历基因重组,使它们容易受到治疗。最近的研究已经产生了一些引人注目的例子,合成致命药物相互作用和生物标志物用于个性化医疗的宏基因组学,通过靶向癌症细胞因原发性功能丧失突变而使用的次要途径,选择性地杀死癌细胞,同时保留健康细胞。发现可操作的表观遗传依赖性和克服肿瘤异质性仍然是将这些迷人的科学发现转化为临床的最大挑战。表观遗传调节剂与基于合成致死性的治疗体系结构的融合将在精确肿瘤学中定义一个变革范例。通过协调染色质调节网络、DNA损伤反应途径和环境特异性脆弱性节点的多层扰动,这种综合策略超越了传统的以靶点为中心的药理学的局限性,并实现了机制上合理的协同抗肿瘤干预。
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来源期刊
CiteScore
6.40
自引率
2.90%
发文量
186
审稿时长
3-8 weeks
期刊介绍: Current Topics in Medicinal Chemistry is a forum for the review of areas of keen and topical interest to medicinal chemists and others in the allied disciplines. Each issue is solely devoted to a specific topic, containing six to nine reviews, which provide the reader a comprehensive survey of that area. A Guest Editor who is an expert in the topic under review, will assemble each issue. The scope of Current Topics in Medicinal Chemistry will cover all areas of medicinal chemistry, including current developments in rational drug design, synthetic chemistry, bioorganic chemistry, high-throughput screening, combinatorial chemistry, compound diversity measurements, drug absorption, drug distribution, metabolism, new and emerging drug targets, natural products, pharmacogenomics, and structure-activity relationships. Medicinal chemistry is a rapidly maturing discipline. The study of how structure and function are related is absolutely essential to understanding the molecular basis of life. Current Topics in Medicinal Chemistry aims to contribute to the growth of scientific knowledge and insight, and facilitate the discovery and development of new therapeutic agents to treat debilitating human disorders. The journal is essential for every medicinal chemist who wishes to be kept informed and up-to-date with the latest and most important advances.
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