Related Experiment Video
Updated: Oct 7, 2025
Using CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells
Published on: July 22, 2019
Targeting Cancer with CRISPR/Cas9-Based Therapy
Katarzyna Balon1, Adam Sheriff2, Joanna Jacków3
1Laboratory of Genomics & Bioinformatics, Hirszfeld Institute of Immunology and Experimental Therapy, Polish Academy of Sciences, 53-114 Wroclaw, Poland.
Abstract:
Cancer is a devastating condition characterised by the uncontrolled division of cells with many forms remaining resistant to current treatment. A hallmark of cancer is the gradual accumulation of somatic mutations which drive tumorigenesis in cancerous cells, creating a mutation landscape distinctive to a cancer type, an individual patient or even a single tumour lesion. Gene editing with CRISPR/Cas9-based tools now enables the precise and permanent targeting of mutations and offers an opportunity to harness this technology to target oncogenic mutations. However, the development of safe and effective gene editing therapies for cancer relies on careful design to spare normal cells and avoid introducing other mutations. This article aims to describe recent advancements in cancer-selective treatments based on the CRISPR/Cas9 system, especially focusing on strategies for targeted delivery of the CRISPR/Cas9 machinery to affected cells, controlling Cas9 expression in tissues of interest and disrupting cancer-specific genes to result in selective death of malignant cells.
Insights
CRISPR/Cas9 gene editing offers a promising approach to target cancer-causing mutations. Advancements focus on safe and effective delivery to cancer cells, aiming for selective tumor cell death while sparing healthy tissues.
Area of Science:
- Oncology
- Molecular Biology
- Gene Therapy
Background:
- Cancer is characterized by uncontrolled cell division and accumulating somatic mutations.
- Many cancer forms are resistant to current treatments.
- CRISPR/Cas9 technology allows precise targeting of genetic mutations.
Purpose of the Study:
- To review recent advancements in CRISPR/Cas9-based cancer-selective treatments.
- To highlight strategies for targeted delivery and controlled expression of CRISPR/Cas9.
- To discuss disrupting cancer-specific genes for selective malignant cell death.
Main Methods:
- Review of recent scientific literature on CRISPR/Cas9 applications in oncology.
- Focus on targeted delivery systems for CRISPR/Cas9 machinery.
- Strategies for controlling Cas9 expression in specific tissues.
- Methods for disrupting oncogenic mutations in cancer cells.
Main Results:
- CRISPR/Cas9 systems are being developed for precise targeting of oncogenic mutations.
- Strategies are emerging for cancer-selective delivery and controlled expression of CRISPR/Cas9.
- Disruption of cancer-specific genes can lead to selective death of malignant cells.
- Careful design is crucial to avoid off-target mutations and spare normal cells.
Conclusions:
- CRISPR/Cas9 gene editing holds significant potential for developing novel cancer therapies.
- Targeted delivery and controlled expression are key challenges for safe and effective clinical application.
- Future research should focus on optimizing these strategies for robust cancer treatment.
Related Concept Videos
01:59CRISPR
01:28CRISPR/Cas9 Genome Editing
02:57Targeted Cancer Therapies
There are several types of targeted therapies against...
02:53CRISPR and crRNAs
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...