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. 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 , P. / Stein, D. / Tang, W. W. / Frank, I. / Wang, S. Q. / Lee, G. / Spratt, S. K. / Surosky, R. T. / Giedling, 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. 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, G. / Fonseca Sales Medeiros, L. / Mitsuo Lima, F. (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.