PMID- 31711197 OWN - NLM STAT- MEDLINE DCOM- 20210719 LR - 20210719 IS - 2041-2657 (Electronic) IS - 2041-2649 (Print) IS - 2041-2649 (Linking) VI - 19 IP - 3 DP - 2020 May 20 TI - CRISPR therapy towards an HIV cure. PG - 201-208 LID - 10.1093/bfgp/elz021 [doi] AB - Tools based on RNA interference (RNAi) and the recently developed clustered regularly short palindromic repeats (CRISPR) system enable the selective modification of gene expression, which also makes them attractive therapeutic reagents for combating HIV infection and other infectious diseases. Several parallels can be drawn between the RNAi and CRISPR-Cas9 platforms. An ideal RNAi or CRISPR-Cas9 therapeutic strategy for treating infectious or genetic diseases should exhibit potency, high specificity and safety. However, therapeutic applications of RNAi and CRISPR-Cas9 have been challenged by several major limitations, some of which can be overcome by optimal design of the therapy or the design of improved reagents. In this review, we will discuss some advantages and limitations of anti-HIV strategies based on RNAi and CRISPR-Cas9 with a focus on the efficiency, specificity, off-target effects and delivery methods. CI - (c) The Author(s) 2019. Published by Oxford University Press. All rights reserved. For Permissions, please email: journals.permissions@oup.com. FAU - Herrera-Carrillo, Elena AU - Herrera-Carrillo E AD - Department of Medical Microbiology Laboratory of Experimental Virology Amsterdam UMC, AMC, University of Amsterdam, Amsterdam, the Netherlands. FAU - Gao, Zongliang AU - Gao Z AD - Department of Medical Microbiology Laboratory of Experimental Virology Amsterdam UMC, AMC, University of Amsterdam, Amsterdam, the Netherlands. FAU - Berkhout, Ben AU - Berkhout B AD - Department of Medical Microbiology Laboratory of Experimental Virology Amsterdam UMC, AMC, University of Amsterdam, Amsterdam, the Netherlands. AD - Department of Life and Environmental Sciences, University of Cagliari, Cagliari, Italy. LA - eng GR - R01 AI145045/AI/NIAID NIH HHS/United States PT - Journal Article PT - Research Support, N.I.H., Extramural PL - England TA - Brief Funct Genomics JT - Briefings in functional genomics JID - 101528229 SB - IM MH - Clustered Regularly Interspaced Short Palindromic Repeats/*genetics MH - Gene Editing/*methods MH - Genetic Therapy/methods MH - HIV Infections/*genetics MH - Humans MH - Lentivirus/genetics PMC - PMC7239311 OTO - NOTNLM OT - CRISPR-Cas OT - HIV OT - RNA interference OT - gene therapy OT - lentiviral vector OT - polymerase III promoter EDAT- 2019/11/12 06:00 MHDA- 2021/07/20 06:00 PMCR- 2020/11/08 CRDT- 2019/11/12 06:00 PHST- 2019/06/19 00:00 [received] PHST- 2019/08/19 00:00 [revised] PHST- 2019/08/21 00:00 [accepted] PHST- 2019/11/12 06:00 [pubmed] PHST- 2021/07/20 06:00 [medline] PHST- 2019/11/12 06:00 [entrez] PHST- 2020/11/08 00:00 [pmc-release] AID - 5614853 [pii] AID - elz021 [pii] AID - 10.1093/bfgp/elz021 [doi] PST - ppublish SO - Brief Funct Genomics. 2020 May 20;19(3):201-208. doi: 10.1093/bfgp/elz021.