Conditional Control of CRISPR/Cas9 Function by Chemically Modified Oligonucleotides DOI Creative Commons

Liangliang Wang,

Yan Liu, Hongjun Song

et al.

Molecules, Journal Year: 2025, Volume and Issue: 30(9), P. 1956 - 1956

Published: April 28, 2025

The CRISPR (clustered regularly interspaced short palindromic repeats) system has emerged as a revolutionary gene-editing tool with immense potential in gene therapy, functional genomics, and beyond. However, achieving precise spatiotemporal control of editing specific cells tissues while effectively mitigating risks, such off-target effects, remains key challenge for its clinical translation. To overcome these limitations, researchers have developed innovative strategies based on chemical modifications oligonucleotides to enhance the precision, efficiency, controllability CRISPR/Cas9-mediated editing. By introducing conditional responsive elements, photosensitive groups, small-molecule units, supramolecular structures, they successfully achieved dose-dependent regulation CRISPR/Cas9 function. This review provides comprehensive overview recent advancements gRNA oligonucleotides, discussing their applications improving specificity, We also highlight challenges associated offer insights into future directions further advance technology.

Language: Английский

Conditional Control of CRISPR/Cas9 Function by Chemically Modified Oligonucleotides DOI Creative Commons

Liangliang Wang,

Yan Liu, Hongjun Song

et al.

Molecules, Journal Year: 2025, Volume and Issue: 30(9), P. 1956 - 1956

Published: April 28, 2025

The CRISPR (clustered regularly interspaced short palindromic repeats) system has emerged as a revolutionary gene-editing tool with immense potential in gene therapy, functional genomics, and beyond. However, achieving precise spatiotemporal control of editing specific cells tissues while effectively mitigating risks, such off-target effects, remains key challenge for its clinical translation. To overcome these limitations, researchers have developed innovative strategies based on chemical modifications oligonucleotides to enhance the precision, efficiency, controllability CRISPR/Cas9-mediated editing. By introducing conditional responsive elements, photosensitive groups, small-molecule units, supramolecular structures, they successfully achieved dose-dependent regulation CRISPR/Cas9 function. This review provides comprehensive overview recent advancements gRNA oligonucleotides, discussing their applications improving specificity, We also highlight challenges associated offer insights into future directions further advance technology.

Language: Английский

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