Related Experiment Video
Updated: Nov 15, 2025
Using CRISPR/Cas9 Gene Editing to Investigate the Oncogenic Activity of Mutant Calreticulin in Cytokine Dependent Hematopoietic Cells
Published on: January 5, 2018
Erratic journey of CRISPR/Cas9 in oncology from bench-work to successful-clinical therapy
1Department of Biochemistry, Era's Lucknow Medical College and hospital, Era University, Lucknow, Uttar Pradesh, India.
Abstract:
CRISPR is a customized molecular scissor, comprising genetic guide made of RNA and an enzyme, Cas9 which snips DNA in simpler, cheaper and more precise way than any other gene editing tools. In recent years CRISPR/Cas has taken the research world by storm being go-to genome editor for potential gene therapy to fix cancer as well as several hereditary disorders. This review explores the literature around the mechanism of Nobel winning CRISPR/Cas9 and its journey from its discovery to various pre-clinical and clinical trials in oncology, focusing mostly on PD-1 knockout CAR-T cell therapy. It also discusses the hurdles and ethical dispute associated with CRISPR, such as unintended on-target and off-target cuts, embryonic germ-line editing. Despite the controversies regarding the safety of this technique, many studies reported promising results on targeting cancer and other diseases using CRISPR/Cas9. Outcomes from the first successful clinical trial showed the beneficial long term effect on genetically modified T-cells in targeting cancer cells which opens the door for CRISPR to be the most preferred technique to help treating cancer and other diseases in future. As far as germ-line editing is concerned, further studies are needed to support the safety of this technique in humans fixing genetic disorders and mutations. Therefore till date only somatic cell editing is ethically approved.
Insights
CRISPR-Cas9 gene editing offers precise DNA modification for treating diseases like cancer. Early clinical trials show promise, especially in CAR-T cell therapy, though ethical considerations for germline editing remain.
Area of Science:
- Biotechnology
- Genetics
- Molecular Biology
Background:
- CRISPR-Cas9 is a revolutionary gene editing tool utilizing RNA guides and Cas9 enzyme for precise DNA modification.
- It has become a leading technology for potential gene therapies targeting cancer and hereditary disorders.
Purpose of the Study:
- To review the mechanism and discovery of CRISPR-Cas9.
- To explore its applications in pre-clinical and clinical oncology trials, particularly PD-1 knockout CAR-T cell therapy.
- To discuss the challenges and ethical debates surrounding CRISPR technology.
Main Methods:
- Literature review of CRISPR-Cas9 mechanism, discovery, and applications.
- Focus on pre-clinical and clinical trials in oncology, specifically CAR-T cell therapy.
- Analysis of ethical considerations, including on-target/off-target effects and germline editing.
Main Results:
- CRISPR-Cas9 demonstrates simpler, cheaper, and more precise DNA editing than other tools.
- Promising results reported in targeting cancer and other diseases.
- First clinical trials show long-term benefits of genetically modified T-cells in cancer treatment.
Conclusions:
- CRISPR-Cas9 holds significant potential as a preferred technique for treating cancer and other diseases.
- Further research is required to ensure the safety of germline editing for genetic disorders.
- Currently, only somatic cell editing is ethically approved.
Related Concept Videos
01:59CRISPR
01:28CRISPR/Cas9 Genome Editing
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...
02:31Homologous Recombination