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Updated: Jun 18, 2026

Yeast As a Chassis for Developing Functional Assays to Study Human P53
Published on: August 4, 2019
Fundamentals and emerging frontiers in p53-targeted drug development
Hui-Deng Long1,2, Ning Zhang3, Wen-Er Wang4
1Department of Pathology, The Affiliated Taizhou People's Hospital of Nanjing Medical University, Taizhou School of Clinical Medicine, Nanjing Medical University, Taizhou, Jiangsu, 225300, China.
Abstract:
As a key tumor suppressor protein, p53 plays a central role in biological processes such as cell cycle regulation, DNA repair, apoptosis, and metabolism. However, p53 gene mutations or functional inactivation are prevalent in over 50% of human cancers, leading to tumorigenesis, development, and drug resistance, making it an important target for anticancer drug development. Currently, p53-targeted therapy faces challenges such as diverse mutation types, insufficient drug specificity, and drug resistance. This article systematically reviews the fundamental theories and cutting-edge advances in the development of p53-targeted drugs. It elaborates on the structure and function of p53 and its mutation-induced carcinogenic mechanisms, then focuses on analyzing the research history and clinical translation status of small-molecule drugs (e.g., APR-246), discusses the application prospects of gene therapy and immunotherapy strategies, and introduces emerging therapies based on CRISPR and PROTAC technologies. By integrating the latest research findings, this article aims to provide theoretical basis and directional guidance for the precise development and clinical translation of p53-targeted drugs.
Insights
The p53 tumor suppressor is crucial for preventing cancer, but its mutations drive over half of human cancers. This review explores novel p53-targeted therapies, including small molecules, gene therapy, and emerging technologies, to overcome drug resistance.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- The p53 protein is a critical tumor suppressor involved in cell cycle regulation, DNA repair, and apoptosis.
- Mutations or inactivation of the p53 gene occur in over 50% of human cancers, promoting tumorigenesis and drug resistance.
- Targeting p53 is a key strategy in anticancer drug development, but faces challenges like diverse mutation types and resistance.
Purpose of the Study:
- To systematically review the fundamental theories and cutting-edge advances in p53-targeted drug development.
- To analyze the research history and clinical translation of small-molecule drugs targeting p53.
- To discuss the prospects of gene therapy, immunotherapy, CRISPR, and PROTAC technologies for p53-targeted cancer treatment.
Main Methods:
- Literature review of p53 structure, function, and mutation-induced carcinogenesis.
- Analysis of small-molecule drugs (e.g., APR-246) and their clinical status.
- Exploration of gene therapy, immunotherapy, CRISPR, and PROTAC technologies for p53-targeted therapies.
Main Results:
- p53 mutations are a significant driver of cancer development and drug resistance.
- Various therapeutic strategies are being developed to target p53, including small molecules, gene therapy, and novel technologies.
- Emerging approaches like CRISPR and PROTAC offer new avenues for precise p53-targeted drug development.
Conclusions:
- Developing effective p53-targeted drugs requires addressing diverse mutation types and overcoming drug resistance.
- Gene therapy, immunotherapy, CRISPR, and PROTAC technologies show promise for future p53-targeted cancer treatments.
- This review provides a theoretical basis and guidance for precise p53-targeted drug development and clinical translation.
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