Related Experiment Video
Updated: May 20, 2025

Using CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells
Published on: July 22, 2019
Comprehensive strategies for constructing efficient CRISPR/Cas based cancer therapy: Target gene selection, sgRNA
Sathishbabu Paranthaman1, Chinnappa A Uthaiah2, Shadab Md3
1Department of Cell Biology and Molecular Genetics, Sri Devaraj Urs Medical College, Sri Devaraj Urs Academy of Higher Education and Research, Tamaka, Kolar 563103, Karnataka, India.
CRISPR/Cas gene editing offers advanced cancer therapy by precisely targeting mutations. This review details the CRISPR/Cas cancer therapy pipeline, addressing challenges like off-target effects for safer, more effective treatments.
Area of Science:
- Genomic Medicine
- Biotechnology
- Oncology
Background:
- Cancer arises from complex genetic and microenvironmental interactions.
- CRISPR/Cas systems enable precise genome editing for cancer research and therapy.
- Effective CRISPR/Cas cancer treatment requires optimizing gene targets and delivery methods.
Purpose of the Study:
- To outline the CRISPR/Cas cancer therapy pipeline.
- To identify strategies for optimizing guide RNAs (gRNAs and sgRNAs).
- To review delivery modalities and current clinical applications.
Main Methods:
- Literature review of CRISPR/Cas technology in cancer therapy.
- Analysis of gene targeting strategies and optimization of gRNAs/sgRNAs.
- Exploration of viral, non-viral, and extracellular vesicle delivery systems.
Main Results:
- CRISPR/Cas technology has significantly advanced cancer modeling and mutation identification.
- Key challenges include off-target effects, specificity, and immunogenicity.
- Various delivery methods are being explored to enhance therapeutic efficacy.
Conclusions:
- A comprehensive pipeline for CRISPR/Cas cancer therapy development is crucial.
- Optimizing gene targets and delivery systems improves precision, effectiveness, and safety.
- Ongoing research and clinical trials highlight the therapeutic potential of CRISPR/Cas in oncology.
Related Concept Videos
CRISPR
Targeted Cancer Therapies
There are several types of targeted therapies against...
CRISPR 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...
Homologous Recombination
What is Genetic Engineering?
Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...

