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CRISPR/Cas9 for cancer treatment: technology, clinical applications and challenges
Xing Cheng1, Shaoyi Fan2, Chengcai Wen3
1Spinal Cord Injury Center, Heidelberg University Hospital, Heidelberg, Germany.
Abstract:
Clustered regularly interspaced short palindromic repeats (CRISPR) is described as RNA mediated adaptive immune system defense, which is naturally found in bacteria and archaea. CRISPR-Cas9 has shown great promise for cancer treatment in cancer immunotherapy, manipulation of cancer genome and epigenome and elimination or inactivation of carcinogenic viral infections. However, many challenges remain to be addressed to increase its efficacy, including off-target effects, editing efficiency, fitness of edited cells, immune response and delivery methods. Here, we explain CRISPR-Cas classification and its general function mechanism for gene editing. Then, we summarize these preclinical CRISPR-Cas9-based therapeutic strategies against cancer. Moreover, the challenges and improvements of CRISPR-Cas9 clinical applications will be discussed.
Insights
Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing shows promise for cancer therapy, including immunotherapy and genome manipulation. Challenges like off-target effects and delivery must be overcome for clinical success.
Area of Science:
- Biotechnology
- Genetics
- Molecular Biology
Background:
- CRISPR-Cas9 is an adaptive immune system in bacteria and archaea.
- It functions as an RNA-guided DNA endonuclease for gene editing.
- CRISPR-Cas9 has potential applications in cancer research and therapy.
Purpose of the Study:
- To explain CRISPR-Cas classification and gene editing mechanisms.
- To summarize preclinical CRISPR-Cas9 therapeutic strategies for cancer.
- To discuss challenges and improvements for clinical CRISPR-Cas9 applications.
Main Methods:
- Review of CRISPR-Cas classification and function.
- Summary of preclinical CRISPR-Cas9 cancer therapy studies.
- Analysis of challenges and advancements in clinical applications.
Main Results:
- CRISPR-Cas9 offers potential for cancer immunotherapy, genome/epigenome manipulation, and viral infection inactivation.
- Key challenges include off-target effects, editing efficiency, cell fitness, immune response, and delivery.
- Preclinical strategies are being developed, but clinical translation requires addressing these hurdles.
Conclusions:
- CRISPR-Cas9 gene editing holds significant therapeutic potential for various cancers.
- Overcoming technical and biological challenges is crucial for successful clinical translation.
- Further research and development are needed to optimize CRISPR-Cas9 for cancer treatment.
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