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CRISPR/Cas9 Delivery System Engineering for Genome Editing in Therapeutic Applications
| Content Provider | MDPI |
|---|---|
| Author | Cheng, Hao Zhang, Feng Ding, Yang |
| Copyright Year | 2021 |
| Description | The clustered regularly interspaced short palindromic repeats (CRISPR)/associated protein 9 (CRISPR/Cas9) systems have emerged as a robust and versatile genome editing platform for gene correction, transcriptional regulation, disease modeling, and nucleic acids imaging. However, the insufficient transfection and off-target risks have seriously hampered the potential biomedical applications of CRISPR/Cas9 technology. Herein, we review the recent progress towards CRISPR/Cas9 system delivery based on viral and non-viral vectors. We summarize the CRISPR/Cas9-inspired clinical trials and analyze the CRISPR/Cas9 delivery technology applied in the trials. The rational-designed non-viral vectors for delivering three typical forms of CRISPR/Cas9 system, including plasmid DNA (pDNA), mRNA, and ribonucleoprotein (RNP, Cas9 protein complexed with gRNA) were highlighted in this review. The vector-derived strategies to tackle the off-target concerns were further discussed. Moreover, we consider the challenges and prospects to realize the clinical potential of CRISPR/Cas9-based genome editing. |
| Starting Page | 1649 |
| e-ISSN | 19994923 |
| DOI | 10.3390/pharmaceutics13101649 |
| Journal | Pharmaceutics |
| Issue Number | 10 |
| Volume Number | 13 |
| Language | English |
| Publisher | MDPI |
| Publisher Date | 2021-10-09 |
| Access Restriction | Open |
| Subject Keyword | Pharmaceutics Crispr/cas9 Genome Editing Site-specific Trafficking Overcome Off-target Risks Therapeutic Applications |
| Content Type | Text |
| Resource Type | Article |
