CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements
This is a spectacular moment for genetics to evolve in genome editing, which encompasses the precise alteration of the cellular DNA sequences within various species. One of the most fascinating genome-editing technologies currently available is Clustered Regularly Interspaced Palindromic Repeats (CR...
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MDPI AG
2022-11-01
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Online Access: | https://www.mdpi.com/2073-4409/11/22/3615 |
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author | Ali Nasrallah Eric Sulpice Farah Kobaisi Xavier Gidrol Walid Rachidi |
author_facet | Ali Nasrallah Eric Sulpice Farah Kobaisi Xavier Gidrol Walid Rachidi |
author_sort | Ali Nasrallah |
collection | DOAJ |
description | This is a spectacular moment for genetics to evolve in genome editing, which encompasses the precise alteration of the cellular DNA sequences within various species. One of the most fascinating genome-editing technologies currently available is Clustered Regularly Interspaced Palindromic Repeats (CRISPR) and its associated protein 9 (CRISPR-Cas9), which have integrated deeply into the research field within a short period due to its effectiveness. It became a standard tool utilized in a broad spectrum of biological and therapeutic applications. Furthermore, reliable disease models are required to improve the quality of healthcare. CRISPR-Cas9 has the potential to diversify our knowledge in genetics by generating cellular models, which can mimic various human diseases to better understand the disease consequences and develop new treatments. Precision in genome editing offered by CRISPR-Cas9 is now paving the way for gene therapy to expand in clinical trials to treat several genetic diseases in a wide range of species. This review article will discuss genome-editing tools: CRISPR-Cas9, Zinc Finger Nucleases (ZFNs), and Transcription Activator-Like Effector Nucleases (TALENs). It will also encompass the importance of CRISPR-Cas9 technology in generating cellular disease models for novel therapeutics, its applications in gene therapy, and challenges with novel strategies to enhance its specificity. |
first_indexed | 2024-03-09T18:25:05Z |
format | Article |
id | doaj.art-150f8862b6684e62916f857e23df46c1 |
institution | Directory Open Access Journal |
issn | 2073-4409 |
language | English |
last_indexed | 2024-03-09T18:25:05Z |
publishDate | 2022-11-01 |
publisher | MDPI AG |
record_format | Article |
series | Cells |
spelling | doaj.art-150f8862b6684e62916f857e23df46c12023-11-24T07:58:15ZengMDPI AGCells2073-44092022-11-011122361510.3390/cells11223615CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest ImprovementsAli Nasrallah0Eric Sulpice1Farah Kobaisi2Xavier Gidrol3Walid Rachidi4Univ. Grenoble Alpes, CEA, INSERM, 38000 Grenoble, FranceUniv. Grenoble Alpes, CEA, INSERM, 38000 Grenoble, FranceUniv. Grenoble Alpes, CEA, INSERM, 38000 Grenoble, FranceUniv. Grenoble Alpes, CEA, INSERM, 38000 Grenoble, FranceUniv. Grenoble Alpes, CEA, INSERM, 38000 Grenoble, FranceThis is a spectacular moment for genetics to evolve in genome editing, which encompasses the precise alteration of the cellular DNA sequences within various species. One of the most fascinating genome-editing technologies currently available is Clustered Regularly Interspaced Palindromic Repeats (CRISPR) and its associated protein 9 (CRISPR-Cas9), which have integrated deeply into the research field within a short period due to its effectiveness. It became a standard tool utilized in a broad spectrum of biological and therapeutic applications. Furthermore, reliable disease models are required to improve the quality of healthcare. CRISPR-Cas9 has the potential to diversify our knowledge in genetics by generating cellular models, which can mimic various human diseases to better understand the disease consequences and develop new treatments. Precision in genome editing offered by CRISPR-Cas9 is now paving the way for gene therapy to expand in clinical trials to treat several genetic diseases in a wide range of species. This review article will discuss genome-editing tools: CRISPR-Cas9, Zinc Finger Nucleases (ZFNs), and Transcription Activator-Like Effector Nucleases (TALENs). It will also encompass the importance of CRISPR-Cas9 technology in generating cellular disease models for novel therapeutics, its applications in gene therapy, and challenges with novel strategies to enhance its specificity.https://www.mdpi.com/2073-4409/11/22/3615CRISPR-Cas9disease modelinggene therapygenome editing |
spellingShingle | Ali Nasrallah Eric Sulpice Farah Kobaisi Xavier Gidrol Walid Rachidi CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements Cells CRISPR-Cas9 disease modeling gene therapy genome editing |
title | CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements |
title_full | CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements |
title_fullStr | CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements |
title_full_unstemmed | CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements |
title_short | CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements |
title_sort | crispr cas9 technology for the creation of biological avatars capable of modeling and treating pathologies from discovery to the latest improvements |
topic | CRISPR-Cas9 disease modeling gene therapy genome editing |
url | https://www.mdpi.com/2073-4409/11/22/3615 |
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