CRISPR therapy towards an HIV cure

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. Sever...

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Bibliographic Details
Published inBriefings in functional genomics Vol. 19; no. 3; pp. 201 - 208
Main Authors Herrera-Carrillo, Elena, Gao, Zongliang, Berkhout, Ben
Format Journal Article
LanguageEnglish
Published England Oxford University Press 20.05.2020
Subjects
Review Paper
CRISPR-Cas
gene therapy
RNA interference
HIV
polymerase III promoter
lentiviral vector
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Summary: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.
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Joint first authorship
ISSN:2041-2657
2041-2649
2041-2657
DOI:10.1093/bfgp/elz021