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Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
Published on: March 10, 2023
Advanced gene editing technologies for oncology mechanisms, applications, and clinical implementation
Soham Bindu1, Archishman Ash1, Koustav Sarkar2
1Department of Biotechnology, School of Bioengineering, SRM Institute of Science and Technology, Chennai, India.
Abstract:
Advanced gene editing tools have transformed oncology by facilitating precise molecular therapies aimed at the hereditary basis of cancer. This thorough study examines the mechanisms, applications, and clinical implementation of advanced genome editing technologies in cancer treatment. This review commences with the molecular principles of genome editing and DNA repair mechanisms, systematically analyzing established technologies such as Zinc Finger Nucleases, Transcription Activator-Like Effector Nucleases, and various CRISPR/Cas systems (Cas9, Cas12, Cas13), in addition to novel advancements including base editors, prime editors, and the PASTE system. Additionally, hybrid platforms such as ARCUS, MegaTALs, and modified recombinases are examined, highlighting their amalgamation with artificial intelligence, biosensors, and synthetic biology concepts. The study outlines significant applications including functional genomics, disease modeling, synthetic lethality screening, and direct therapeutic interventions, with a specific focus on CAR-T cell engineering and immune checkpoint regulation. Applications unique to various cancer types are thoroughly examined throughout lung, breast, colorectal, hematologic, liver, pancreatic, head & neck, esophageal, prostate, gastric, and brain cancers. Significant obstacles such as delivery optimization via viral and non-viral vectors, tumor-specific targeting, off-target effects, immunogenicity, and ethical issues related to germline vs somatic editing are comprehensively examined. The translational landscape is analyzed via current clinical trials, regulatory structures, and the incorporation of organoid models and patient-derived xenografts for the advancement of personalized therapies. This review highlights the transformative impact of gene editing on cancer medicine, advancing toward more accurate, effective, and personalized therapeutic approaches.
Insights
Advanced gene editing technologies like CRISPR offer precise cancer therapies by targeting the hereditary basis of cancer. This review explores their mechanisms, applications, and challenges in clinical oncology.
Area of Science:
- Oncology and Genetics
- Molecular Biology and Biotechnology
Background:
- Gene editing tools revolutionize oncology by enabling targeted therapies for hereditary cancers.
- Understanding DNA repair mechanisms is crucial for advancing genome editing applications.
Purpose of the Study:
- To comprehensively review advanced genome editing technologies for cancer treatment.
- To analyze their mechanisms, applications, challenges, and translational landscape.
Main Methods:
- Systematic analysis of established (ZFNs, TALENs, CRISPR/Cas) and novel (base editors, prime editors, PASTE) gene editing systems.
- Examination of hybrid platforms (ARCUS, MegaTALs) integrated with AI and synthetic biology.
- Review of applications in functional genomics, disease modeling, and direct therapeutic interventions (e.g., CAR-T cell engineering).
Main Results:
- Detailed examination of gene editing applications across diverse cancer types (lung, breast, colorectal, etc.).
- Identification of key challenges including delivery, targeting, off-target effects, immunogenicity, and ethical considerations.
- Analysis of the translational landscape, clinical trials, regulatory aspects, and use of organoid/xenograft models.
Conclusions:
- Gene editing is transforming cancer medicine, paving the way for more precise and effective personalized therapies.
- Overcoming delivery and safety challenges is critical for clinical translation.
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