Therapeutic Applications of CRISPR/Cas9    

Without a doubt, CRISPR/Cas9 is one of the hottest topics in the biomedical research community today. CRISPR/Cas9 is not only widely adopted in basic research, but well-known pharmaceutical companies and emerging biotechnology companies are racing to develop CRISPR-based therapies. Compared with other gene therapy strategies, CRISPR genome editing is considered faster, cheaper, and safer.

• In genetic diseases

So far, CRISPR/Cas9 has successfully cured genetic diseases in animal models. For example, transcriptionally activated CRISPR/Cas9 with short dgRNA (~ 14 or ~ 15 bp) successfully relieved the disease phenotype in mouse models of type 1 diabetes, acute kidney injury, and muscular dystrophy. Similarly, CRISPR-mediated genome editing has partially improved the symptoms of amyotrophic lateral sclerosis and the SOD1 linked form of autosomal dominant hereditary loss. These advances provide promising insights into the ultimate translation of CRISPR gene editing into the clinic.

• Cancer treatment

The first clinical trial of CRISPR for cancer treatment enrolled its first patient at the West China Hospital of Sichuan University in Sichuan in 2016.

The CRISPR/Cas9 system can be used to promote the production of therapeutic immune cells, including the construction of chimeric antigen receptors (CAR) and T cell receptor (TCR) -T cells. The combination of CAR modification, immune checkpoint suppression, and CRISPR/Cas9 technology is a therapeutic strategy with great potential for treating solid tumors. In addition to modifying immune cells to treat tumors, CRISPR/Cas9 technology can also be used to disrupt sequences that cause viral diseases, such as human papillomavirus (HPV) 16 and HPV18 E6/E7 DNA to prevent virus-related cancers.

• In HIV-1/AIDS Therapy

CRISPR/Cas9 can be used to target cellular cofactors or the HIV-1 genome to reduce HIV-1 infection and clear the provirus, as well as induce transcriptional activation of the latent virus in the latent virus bank to eliminate. This universal gene editing technology has been successfully applied to HIV-1/AIDS prevention and reduction in human cells and animal models

• Limitations of CRISPR/Cas9 applications

➢ A major problem is the potential off-target effect, which may induce important genetic mutations and chromosomal translocations. In clinical applications, reducing off-target effects is always the most important.
➢ It has been shown that the application of RNP in certain types of cells can trigger an innate immune response, resulting in cytotoxicity of the cells. Before conducting clinical trials, the immunogenicity of sgRNA and Cas9 should be evaluated.
➢ Another major challenge is how to effectively deliver this large complex to HIV-1 infected cells.
➢ In addition, social and ethical concerns regarding the application of CRISPR/Cas9 technology deserve public consideration.

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