Medical applications of clustered regularly interspaced short palindromic repeats (CRISPR/Cas) tool: A comprehensive overview

Araldi, Rodrigo Pinheiro; Khalil, Charbel; Grignet, Pedro Henrique; Teixeira, Michelli Ramires; Melo, Thatiana Corrêa de; Módolo, Diego Grando; Fernandes, Luis G. V.; Ruiz, Jorge L.M.; Souza, Edislane Barreiros de

doi:10.1016/j.gene.2020.144636

Cited by 20 publications

(11 citation statements)

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“…CRISPR/Cas9 gene editing technology now allows alterations of several thousand base pairs of nucleotide sequence, revolutionizing the field ( Araldi et al., 2020 ). In LCA10 patients, IVS26 mutations of CEP290 have been successfully repaired by CRISPR/Cas9 technology ( Ruan et al., 2017 ).…”

Section: Gene Therapy For Ocular Diseasesmentioning

confidence: 99%

The Primary Cilium as a Therapeutic Target in Ocular Diseases

Zhou

2020

Front. Pharmacol.

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Primary cilia are microtubule-based cellular structures located on the surfaces of most mammalian cells and play important roles in detecting external stimuli, signal transduction, and cell cycle regulation. Primary cilia are also present in several structures of the eye, and their abnormal development or dysfunction can cause various ocular diseases. The rapid development of proteomics and metabolomics technologies have helped in the identification of many ocular disease-related proteins, some of which are dysregulated in primary cilia. This review focuses on ciliary dysregulation in a number of ocular diseases and discusses the potential of targeting primary cilia in gene and stem cell therapy for these diseases.

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Section: Gene Therapy For Ocular Diseasesmentioning

confidence: 99%

The Primary Cilium as a Therapeutic Target in Ocular Diseases

Zhou

2020

Front. Pharmacol.

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“…TALENs have quickly established themselves as a viable alternative to ZFNs for genome editing. Although it is based on a non-specific FokI nuclease domain that may be fused to a customized DNA-binding domain, it differs from ZFNs in that the DNA binding domain is made up of highly conserved repeats derived from TALEs (proteins secreted by Xanthomonas bacteria) [ 15 , 16 ].…”

Section: Introductionmentioning

confidence: 99%

From Descriptive to Functional Genomics of Leukemias Focusing on Genome Engineering Techniques

Balla

Tripon

Bănescu

2021

IJMS

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Genome engineering makes the precise manipulation of DNA sequences possible in a cell. Therefore, it is essential for understanding gene function. Meganucleases were the start of genome engineering, and it continued with the discovery of Zinc finger nucleases (ZFNs), followed by Transcription activator-like effector nucleases (TALENs). They can generate double-strand breaks at a desired target site in the genome, and therefore can be used to knock in mutations or knock out genes in the same way. Years later, genome engineering was transformed by the discovery of clustered regularly interspaced short palindromic repeats (CRISPR). Implementation of CRISPR systems involves recognition guided by RNA and the precise cleaving of DNA molecules. This property proves its utility in epigenetics and genome engineering. CRISPR has been and is being continuously successfully used to model mutations in leukemic cell lines and control gene expression. Furthermore, it is used to identify targets and discover drugs for immune therapies. The descriptive and functional genomics of leukemias is discussed in this study, with an emphasis on genome engineering methods. The CRISPR/Cas9 system’s challenges, viewpoints, limits, and solutions are also explored.

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“…The genome modifying mechanism CRISPR (clustered regularly interspaced short palindromic repeats) lets scientists edit specific sequences of DNA 1 and provides a new strategy for the development of precision treatments for a wide range of diseases. 2 The CRISPR-Cas system utilizes short guide RNAs (sgRNAs) to direct site-specific binding of the endonuclease Cas9. The 2 cleavage domains of Cas9 catalyze a double-stranded DNA break.…”

Section: Commentarymentioning

confidence: 99%