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Updated: Jan 30, 2026

Genome Engineering of Primary Human B Cells Using CRISPR/Cas9
Published on: November 3, 2020
CRISPR-Cas9 a boon or bane: the bumpy road ahead to cancer therapeutics
Debarati Ghosh1, Prabhadevi Venkataramani1, Saikat Nandi1
1Cold Spring Harbor Laboratory, Cold Spring Harbor, NY USA.
Abstract:
Genome editing allows for the precise manipulation of DNA sequences in a cell making this technology essential for understanding gene function. CRISPR/Cas9 is a targeted genome-editing platform derived from bacterial adaptive immune system and has been repurposed into a genome-editing tool. The RNA-guided DNA endonuclease, Cas9 can be easily programmed to target new sites by altering its guide RNA sequence, making this technology easier, more efficient, scalable and an indispensable tool in biological research. This technology has helped genetically engineer animal models to understand disease mechanisms and elucidate molecular details that can be exploited for improved therapeutic outcomes. In this review, we describe the CRISPR-Cas9 gene-editing mechanism, CRISPR-screening methods, therapeutic targeting of CRISPR in animal models and in cancer immunotherapy. We also discuss the ongoing clinical trials using this tool, limitations of this tool that might impede the clinical applicability of CRISPR-Cas9 and future directions for developing effective CRISPR-Cas9 delivery systems that may improve cancer therapeutics.
Insights
CRISPR-Cas9 gene editing precisely modifies DNA for biological research and disease modeling. This review covers its mechanism, applications in animal models, cancer immunotherapy, and future therapeutic delivery systems.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- Genome editing enables precise DNA manipulation, crucial for understanding gene function.
- CRISPR/Cas9, derived from bacterial immunity, is a versatile RNA-guided DNA endonuclease.
- Its programmability makes it efficient, scalable, and indispensable for biological research.
Purpose of the Study:
- To review the CRISPR-Cas9 gene-editing mechanism.
- To explore CRISPR-screening methods and therapeutic applications.
- To discuss clinical trials, limitations, and future directions for CRISPR-Cas9.
Main Methods:
- Review of CRISPR-Cas9 gene-editing mechanism.
- Analysis of CRISPR-screening methodologies.
- Examination of therapeutic targeting in animal models and cancer immunotherapy.
Main Results:
- CRISPR-Cas9 facilitates genetic engineering of animal models for disease mechanism studies.
- It aids in elucidating molecular details for improved therapeutic outcomes.
- The technology shows promise in cancer immunotherapy and ongoing clinical trials.
Conclusions:
- CRISPR-Cas9 is a powerful tool for biological research and therapeutic development.
- Ongoing research focuses on overcoming limitations for clinical applicability.
- Developing effective delivery systems is key for advancing CRISPR-Cas9 in cancer therapeutics.
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