2021
DOI: 10.1007/s11248-020-00232-9
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Mini review: genome and transcriptome editing using CRISPR-cas systems for haematological malignancy gene therapy

Abstract: The recent introduction of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR associated protein (Cas) systems, offer an array of genome and transcriptome editing tools for clinical repair strategies. These include Cas9, Cas12a, dCas9 and more recently Cas13 effectors. RNA targeting CRISPR-Cas13 complexes show unique characteristics with the capability to engineer transcriptomes and modify gene expression, providing a potential clinical cancer therapy tool across various tissue types… Show more

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Cited by 5 publications
(3 citation statements)
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References 59 publications
(87 reference statements)
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“…Recent computational efforts to identify new CRISPR systems uncovered a novel type of RNA targeting enzyme, Cas13. The diverse Cas13 family contains at least four known subtypes, including Cas13a, Cas13b, Cas13c, and Cas13d (Elliott et al, 2021). One of the most straightforward applications of Cas13 in vivo is targeted RNA knockdown using mammalian codon optimized Cas13 and guide expression vectors.…”
Section: Cas13 Systemmentioning
confidence: 99%
“…Recent computational efforts to identify new CRISPR systems uncovered a novel type of RNA targeting enzyme, Cas13. The diverse Cas13 family contains at least four known subtypes, including Cas13a, Cas13b, Cas13c, and Cas13d (Elliott et al, 2021). One of the most straightforward applications of Cas13 in vivo is targeted RNA knockdown using mammalian codon optimized Cas13 and guide expression vectors.…”
Section: Cas13 Systemmentioning
confidence: 99%
“…According to the diversity of cas genes and the mechanism of action for Cas proteins, CRISPR-Cas systems are categorized into 2 classes and further classified into six different types: type I to type VI [7,8]. Basic CRISPR research only constitutes a relatively small portion, and the majority of work rather focuses on the application of CRISPR-Cas systems in genome-editing, transcriptional control, biotechnology, and agriculture engineering [9][10][11][12][13][14][15]. Furthermore, how CRISPR-Cas systems interact with other genes in the prokaryote, such as endogenous gene targeting and virulence gene regulation, remains largely un-elucidated [16][17][18][19][20].…”
Section: Introductionmentioning
confidence: 99%
“…Clustered regularly interspaced short palindromic repeats/ CRISPR associated nuclease (CRISPR/Cas) systems open new horizons for biosensors, for instance, CRISPR/Cas9, 15,16 CRISPR/ Cas12a, 17,18 CRISPR/Cas13a, [19][20][21] and CRISPR/Cas14a. [22][23][24] Among them, CRISPR/Cas12a and CRISPR/Cas13a are highly promising candidates for detecting DNA and RNA due to their trans-cleavage activity, which is the basis of signal amplification.…”
mentioning
confidence: 99%