CRISPR/Cas9 gene therapy against AIDS

Various experiments on animal models aimed at combating the Human Immunodeficiency Virus (HIV) using the CRISPR/Cas9 technique have been presented in recent years. A team of researchers from Temple University in Philadelphia and the University of Pittsburgh reported in May 2017 on a study in a mouse model in which the researchers succeeded in removing the viral genome of the HI virus from 95% of infected mouse cells using the CRISPR/Cas9 technique. 

Combating HIV using the other so-called genetic scissors, zinc finger nuclease (ZNF), has already been investigated in humans in a clinical study. During this study, the team of researchers succeeded in making some of the patients' immune cells resistant to the HI virus by suppressing a specific gene.

On the CRISPR/Cas9 gene therapy study:

Yin, Chaoran / Zhang, Ting / Qu, Xiying / Zhang, Yonggang / Putatunda, Raj / Xiao, Xiao / Li, Fang / Xiao, Weidong / Zhao, Huaqing / Dai, Shen / Qin, Xuebin / Mo, Xianming / Young, Won-Bin / Khalili, Kamel / Hu, Wenhui (2017): In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models. In: Molecular Therapy 25(5), 1168–1186. doi: 10.1016/j.ymthe.2017.03.012 Online Version

On the study of the treatment of HIV using zinc finger nucleases:

Tebas , Pablo / Stein, David / Tang, Winston W. / Frank, Ian / Wang, Shelley Q. / Lee, Gary / Spratt, S. Kaye / Surosky, Richard T. / Giedling, Martin A. / Nichol, Geoff / Holmes, Michael C. / Gregory, Philip D. / Ando, Dale G. / Kalos, Micheal / Collman, Ronald G. / Binder-Scholl, Gwendolyn / Plesa, Gabriela / Hwang, Wie-Ting / Levine, Bruce L. / June, Carl H. (2014): Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV. In: The New England Journal of Medicine 370, 901–910. doi: 10.1056/NEJMoa1300662 Online Version 

An overview of possible applications of CRISPR/Cas in the treatment of HIV:

De Nardi Sanches-da-Silva, Gabriela / Fonseca Sales Medeiros, Luiza / Mitsuo Lima, Fabio (2019): The Potential Use of the CRISPR-Cas System for HIV-1 Gene Therapy. In: International Journal of Genomics. doi: 10.1155/2019/8458263 Online Version 

Further information:

In the module Genome Editing in this focus, the ZFN and CRISPR/Cas9-procedures are explained.

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