Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing

Mengstie, Misganaw Asmamaw; Zawdie, Belay

doi:10.2147/btt.s326422

Cited by 117 publications

(70 citation statements)

References 72 publications

(83 reference statements)

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“…Cas9 activates and using RuvC-like and HNH domains makes two nicks in two complementary strands at the target locus, resulting in a DSB ( Jinek et al, 2012 ). The CRISPR/Cas9 system has opened up numerous opportunities for genome editing in different organisms, and now there are many reports on its various applications [for review see ( Mengstie and Wondimu, 2021 )]; in particular, this system is used to create animal models of human diseases ( Leonova and Gainetdinov, 2020 ). It has found many applications in biotechnology, including cracking the challenge of antibiotic resistance ( Matharu et al, 2019 ; Novick, 2021 , 202; Zohra et al, 2021 ).…”

Section: Therapy For Genetic Diseasesmentioning

confidence: 99%

Translational potential of base-editing tools for gene therapy of monogenic diseases

Reshetnikov

Chirinskaite

Sopova

et al. 2022

Front. Bioeng. Biotechnol.

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Millions of people worldwide have rare genetic diseases that are caused by various mutations in DNA sequence. Classic treatments of rare genetic diseases are often ineffective, and therefore great hopes are placed on gene-editing methods. A DNA base–editing system based on nCas9 (Cas9 with a nickase activity) or dCas9 (a catalytically inactive DNA-targeting Cas9 enzyme) enables editing without double-strand breaks. These tools are constantly being improved, which increases their potential usefulness for therapies. In this review, we describe the main types of base-editing systems and their application to the treatment of monogenic diseases in experiments in vitro and in vivo. Additionally, to understand the therapeutic potential of these systems, the advantages and disadvantages of base-editing systems are examined.

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Section: Therapy For Genetic Diseasesmentioning

confidence: 99%

Translational potential of base-editing tools for gene therapy of monogenic diseases

Reshetnikov

Chirinskaite

Sopova

et al. 2022

Front. Bioeng. Biotechnol.

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“…Nearly 80% of the human population is seropositive for AAVs, and to date, they are not linked to any type of human disease. Because of their low cytotoxicity, immunogenicity, and the very low chance of integration into the human genome, it is currently at the top of the list of leading in vivo delivery systems for the CRISPR components compared to other viral vectors [ 106 , 107 ]. Another key advantage is the presence of several well-known AAV serotypes for a specific and different tissue tropism, such as for heart cells, epithelial lung cells, neurons, and skeletal muscle cells, making them very efficient for an effective directed tissue-specific delivery for in vivo genome editing [ 108 ].…”

Section: Disease Modeling and Gene Therapy Using Crispr-cas9mentioning

confidence: 99%

CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements

Nasrallah

Sulpice

Kobaisi

et al. 2022

Cells

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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.

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“…Looking into the history, in 1987, a researcher Ishino and his group from Japan serendipitously recognized short repetitive sequences in Escherichia coli while identifying certain genes liable for conversion of ALP isozyme [8]. But they did not unveil any of its biological significance.…”

Section: History and Evolutionmentioning

confidence: 99%

“…Thus, new ideas and techniques are required to increase natural-food production. Here, CRISPR-Cas which is an existing system, is being used to modify crops and foods to enhance their various properties such as nutritional values, shelf-life, disease resistance and drought-tolerance [8,34]. Overall, CRISPR can help to solve food crisis in three common ways.…”

Section: Agriculturementioning

confidence: 99%

Overview of CRISPR-Cas Mediated Genome Engineering

Bhuvana

Ranjitha²

2022

JALSI

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Genetic diseases are prevailing in 3-5% of new-born worldwide and estimated to cause around 50% of child deaths. The development of efficient and regulated system for customizing genome modifications to treat such disorders has been the zone of interest. Over time, different editing techniques have emerged based on programmable nucleases such as ZFN or TALENS but among all, CRISPR-Cas has significantly progressed as alteration tool from bench to clinical practices. CRISPR-Cas is an immune system discovered in prokaryotes that enables organism to recognize and destroy any invading genetic elements. This functional property of CRISPR has opened up plethora of applications across different disciplines such as diagnostics, agriculture and therapeutics. So here, the review attempt to discuss origin, mechanisms pertinent to CRISPR and applications along with challenges concerning them.

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Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing

Cited by 117 publications

References 72 publications

Translational potential of base-editing tools for gene therapy of monogenic diseases

Translational potential of base-editing tools for gene therapy of monogenic diseases

CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements

Overview of CRISPR-Cas Mediated Genome Engineering

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