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, C., Zhang, T., Qu, X., Zhang, Y., Putatunda, R., Xiao, X., Li, F., Xiao, W., Zhao, H., Dai, S., Qin, X., Mo, X., Young, W. B., Khalili, K., & Hu, W. (2017). In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models. Molecular therapy: the journal of the American Society of Gene Therapy, 25(5), 1168–1186. https://doi.org/10.1016/j.ymthe.2017.03.012
On the study of the treatment of HIV using zinc finger nucleases:
Tebas, P., Stein, D., Tang, W. W., Frank, I., Wang, S. Q., Lee, G., Spratt, S. K., Surosky, R. T., Giedlin, M. A., Nichol, G., Holmes, M. C., Gregory, P. D., Ando, D. G., Kalos, M., Collman, R. G., Binder-Scholl, G., Plesa, G., Hwang, W. T., Levine, B. L., & June, C. H. (2014). Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. The New England journal of medicine, 370(10), 901–910. https://doi.org/10.1056/NEJMoa1300662
An overview of possible applications of CRISPR/Cas in the treatment of HIV:
Sanches-da-Silva, G. N., Medeiros, L. F. S., & Lima, F. M. (2019). The Potential Use of the CRISPR-Cas System for HIV-1 Gene Therapy. International journal of genomics, 2019, 8458263. https://doi.org/10.1155/2019/8458263
Further information:
In the module Genome Editing in this focus, the ZFN and CRISPR/Cas9-procedures are explained.