The application and therapeutic potential of CRISPR/Cas9 technology in diseases

Tongzheng Yao

doi:10.54097/b54ky443

Authors

Tongzheng Yao

DOI:

https://doi.org/10.54097/b54ky443

Keywords:

CRISPR/Cas9 technology; HIV/AIDS; Duchenne Muscular Dystrophy (DMD); Off-target effect.

Abstract

The application of CRISPR technology is one of the focuses of current research orientation. At present, CRISPR technology has reached a new level, but there are still some challenges in the clinical application of human diseases. This paper introduces the background and principle of CRISPR technology and cites two specific diseases for case analysis. It discusses the feasibility, limitations and possible solutions of CRISPR technology in the clinical treatment of actual diseases. Studies have found that CRISPR technology has the ability to cut HIV virus genes that have been integrated into the host genome, which is expected to eliminate the potential viral reservoir to achieve the goal of complete cure. In the treatment of DMD, CRISPR technology can restore the expression of dystrophin, so that the patient can be completely cured. However, safety evaluation is still needed before practical clinical application. Finding alternative vector materials, editing the components of CRISPR system, and maintaining long-term efficacy have become the main problems to be solved in practical applications.

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