TY - JOUR
T1 - CRISPR therapy towards an HIV cure
AU - Herrera-Carrillo, Elena
AU - Gao, Zongliang
AU - Berkhout, Ben
N1 - Publisher Copyright: © The Author(s) 2019. Copyright: Copyright 2020 Elsevier B.V., All rights reserved.
PY - 2020/5/1
Y1 - 2020/5/1
N2 - 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.
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.
KW - CRISPR-Cas
KW - Gene therapy
KW - HIV
KW - Lentiviral vector
KW - Polymerase III promoter
KW - RNA interference
UR - http://www.scopus.com/inward/record.url?scp=85085232715&partnerID=8YFLogxK
U2 - https://doi.org/10.1093/bfgp/elz021
DO - https://doi.org/10.1093/bfgp/elz021
M3 - Article
C2 - 31711197
SN - 2041-2657
VL - 19
SP - 201
EP - 208
JO - Briefings in functional genomics
JF - Briefings in functional genomics
IS - 3
ER -