Gene Editing Using CRISPR/Cas9 System in the Treatment of HIV and Cancers

Abstract

CRISPR/Cas9 has proved its revolutionary power in treating difficult diseases (such as HIV and cancer) with fresh horizons for precise medicine. CRISPR/Cas9 as a powerful tool are currently under investigation in the fields of HIV cure, for its ability to knock-out viral genome by editing host cell genes through disrupting the viral reservoir. In the cancer therapy, its aims are to boost the anti-tumor action of the immune cells (e.g. CAR-T cells) or direct the cancer gene mutation site to make the cancer cell apoptosis, but its off-target effect, in vivo delivery efficiency and the problem of longterm safety also are needed to solve. In this paper, we systematically review CRISPR/Cas9 research in HIV antiviral immune activation, the engineering on cancer immune cells, and tumor genome editing. It revises its performance in in vitro cells and animals and the technical challenges it confronts and points out the targeting capability and editing efficiency of the delivery system as two key factors determining its clinical translation. The study gives a theoretical guide for optimization of gene editing strategy which might be helpful for the development of safe and efficient gene therapy. This also underscores the necessity for future research to focus upon the design of smart delivery vectors, the generation of multi-editing combinatorial therapies and the long-term toxicity studies – all of which urgently need further interdisciplinary efforts to enable this technology to be translated from the lab, to the clinic.