Optimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal Cell

During recent years, clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein 9 (Cas9) technologies have been noticed as a rapidly evolving tool to deliver a possibility for modifying target sequence expression and function. The CRISPR/Cas9 tool is currently being...

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Autores principales:	Sadegh Shojaei Baghini, Zhanna R. Gardanova, Angelina Olegovna Zekiy, Navid Shomali, Foad Tosan, Mostafa Jarahian
Formato:	article
Lenguaje:	EN
Publicado:	Frontiers Media S.A. 2021
Materias:	CRISPR/Cas9 tool genome-editing SgRNA off-target effects targeted therapy Biotechnology TP248.13-248.65
Acceso en línea:	https://doaj.org/article/a3d9d865b9854b8b8907d754bfdda61b
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spelling	oai:doaj.org-article:a3d9d865b9854b8b8907d754bfdda61b2021-11-19T07:12:41ZOptimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal Cell2296-418510.3389/fbioe.2021.775309https://doaj.org/article/a3d9d865b9854b8b8907d754bfdda61b2021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fbioe.2021.775309/fullhttps://doaj.org/toc/2296-4185During recent years, clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein 9 (Cas9) technologies have been noticed as a rapidly evolving tool to deliver a possibility for modifying target sequence expression and function. The CRISPR/Cas9 tool is currently being used to treat a myriad of human disorders, ranging from genetic diseases and infections to cancers. Preliminary reports have shown that CRISPR technology could result in valued consequences for the treatment of Duchenne muscular dystrophy (DMD), cystic fibrosis (CF), β-thalassemia, Huntington’s diseases (HD), etc. Nonetheless, high rates of off-target effects may hinder its application in clinics. Thereby, recent studies have focused on the finding of the novel strategies to ameliorate these off-target effects and thereby lead to a high rate of fidelity and accuracy in human, animals, prokaryotes, and also plants. Meanwhile, there is clear evidence indicating that the design of the specific sgRNA with high efficiency is of paramount importance. Correspondingly, elucidation of the principal parameters that contributed to determining the sgRNA efficiencies is a prerequisite. Herein, we will deliver an overview regarding the therapeutic application of CRISPR technology to treat human disorders. More importantly, we will discuss the potent influential parameters (e.g., sgRNA structure and feature) implicated in affecting the sgRNA efficacy in CRISPR/Cas9 technology, with special concentration on human and animal studies.Sadegh Shojaei BaghiniZhanna R. GardanovaZhanna R. GardanovaAngelina Olegovna ZekiyNavid ShomaliFoad TosanMostafa JarahianFrontiers Media S.A.articleCRISPR/Cas9 toolgenome-editingSgRNAoff-target effectstargeted therapyBiotechnologyTP248.13-248.65ENFrontiers in Bioengineering and Biotechnology, Vol 9 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	CRISPR/Cas9 tool genome-editing SgRNA off-target effects targeted therapy Biotechnology TP248.13-248.65
spellingShingle	CRISPR/Cas9 tool genome-editing SgRNA off-target effects targeted therapy Biotechnology TP248.13-248.65 Sadegh Shojaei Baghini Zhanna R. Gardanova Zhanna R. Gardanova Angelina Olegovna Zekiy Navid Shomali Foad Tosan Mostafa Jarahian Optimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal Cell
description	During recent years, clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein 9 (Cas9) technologies have been noticed as a rapidly evolving tool to deliver a possibility for modifying target sequence expression and function. The CRISPR/Cas9 tool is currently being used to treat a myriad of human disorders, ranging from genetic diseases and infections to cancers. Preliminary reports have shown that CRISPR technology could result in valued consequences for the treatment of Duchenne muscular dystrophy (DMD), cystic fibrosis (CF), β-thalassemia, Huntington’s diseases (HD), etc. Nonetheless, high rates of off-target effects may hinder its application in clinics. Thereby, recent studies have focused on the finding of the novel strategies to ameliorate these off-target effects and thereby lead to a high rate of fidelity and accuracy in human, animals, prokaryotes, and also plants. Meanwhile, there is clear evidence indicating that the design of the specific sgRNA with high efficiency is of paramount importance. Correspondingly, elucidation of the principal parameters that contributed to determining the sgRNA efficiencies is a prerequisite. Herein, we will deliver an overview regarding the therapeutic application of CRISPR technology to treat human disorders. More importantly, we will discuss the potent influential parameters (e.g., sgRNA structure and feature) implicated in affecting the sgRNA efficacy in CRISPR/Cas9 technology, with special concentration on human and animal studies.
format	article
author	Sadegh Shojaei Baghini Zhanna R. Gardanova Zhanna R. Gardanova Angelina Olegovna Zekiy Navid Shomali Foad Tosan Mostafa Jarahian
author_facet	Sadegh Shojaei Baghini Zhanna R. Gardanova Zhanna R. Gardanova Angelina Olegovna Zekiy Navid Shomali Foad Tosan Mostafa Jarahian
author_sort	Sadegh Shojaei Baghini
title	Optimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal Cell
title_short	Optimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal Cell
title_full	Optimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal Cell
title_fullStr	Optimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal Cell
title_full_unstemmed	Optimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal Cell
title_sort	optimizing sgrna to improve crispr/cas9 knockout efficiency: special focus on human and animal cell
publisher	Frontiers Media S.A.
publishDate	2021
url	https://doaj.org/article/a3d9d865b9854b8b8907d754bfdda61b
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_version_	1718420331537891328

Optimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal Cell

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