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CRISPR/Cas9 Delivery System Engineering for Genome Editing in Therapeutic Applications
Hao Cheng1,2,3, Feng Zhang1,2,3, Yang Ding1,2,3
1Key Laboratory of Drug Quality Control and Pharmacovigilance, China Pharmaceutical University, Ministry of Education, Nanjing 210009, China.
Pharmaceutics
|October 23, 2021
Summary
CRISPR/Cas9 genome editing shows promise but faces delivery challenges. This review covers viral and non-viral vectors, clinical trials, and strategies to improve CRISPR/Cas9 delivery and reduce off-target effects for therapeutic applications.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- CRISPR/Cas9 is a powerful genome editing tool with applications in gene correction, transcriptional regulation, and disease modeling.
- Key limitations hindering CRISPR/Cas9's clinical translation include inefficient delivery and off-target effects.
Purpose of the Study:
- To review advancements in CRISPR/Cas9 system delivery methods, focusing on viral and non-viral vectors.
- To summarize and analyze CRISPR/Cas9 delivery technologies used in ongoing clinical trials.
- To discuss strategies for mitigating off-target effects and realizing the therapeutic potential of CRISPR/Cas9.
Main Methods:
- Review of current literature on CRISPR/Cas9 delivery systems.
- Analysis of viral and non-viral vector strategies for delivering CRISPR/Cas9 components (pDNA, mRNA, RNP).
- Examination of clinical trial data and associated delivery technologies.
Main Results:
- Progress in CRISPR/Cas9 delivery using both viral and non-viral vectors is detailed.
- Non-viral vectors, including rationally designed options for pDNA, mRNA, and RNP delivery, are highlighted.
- Strategies to address off-target concerns associated with vector delivery are discussed.
Conclusions:
- Optimizing CRISPR/Cas9 delivery is crucial for overcoming current limitations.
- Further research into advanced vector systems and off-target mitigation is needed to fully realize CRISPR/Cas9's clinical potential.
- Successful clinical translation of CRISPR/Cas9 genome editing relies on effective and safe delivery strategies.
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