2014
DOI: 10.1172/jci72992
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Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9

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Cited by 402 publications
(311 citation statements)
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“…Homologous recombination of exogenously supplied sequences can result in genetic modiications (knock-ins). CRISPR technology has important advantages over TALENS and ZFNs including; ease of use [164] target site selection [165] and overall eiciency, although of-target efects remains an important issue of concern [166]. CRISPR/Cas9 system may be used to alter the expression of resistance genes for constitutive expression against plant-parasitic nematodes.…”
Section: Host Resistancementioning
confidence: 99%
“…Homologous recombination of exogenously supplied sequences can result in genetic modiications (knock-ins). CRISPR technology has important advantages over TALENS and ZFNs including; ease of use [164] target site selection [165] and overall eiciency, although of-target efects remains an important issue of concern [166]. CRISPR/Cas9 system may be used to alter the expression of resistance genes for constitutive expression against plant-parasitic nematodes.…”
Section: Host Resistancementioning
confidence: 99%
“…The effect can act on the whole organism, in target tissues or cell types permanently, or under specific conditions. In recent years, the gene manipulation tools have been extended by gene editing, a protocol that targets single nucleotides or short DNA sequences and allows subtle or extreme, deleterious or advantageous modifications of DNA to study such effects on genes, proteins, or regulatory elements (Gupta and Musunuru, 2014). , 2012).…”
Section: Tools For Functional Tests Of Genesmentioning
confidence: 99%
“…This permits targeted analyses of functional domains of a gene product as well as the regulation of a gene (Esvelt et al, 2013;Gupta and Musunuru, 2014). Effective protocols that exist for gene editing use guide RNA in combination with the enzyme recombinase (CRISPR/Cas9), zinc finger nuclease, or zinc finger protein in combination with a nuclease (TALEN) (Gupta and Musunuru, 2014). Although these methods can be applied easily to livestock species, the mouse still has the unequal advantage of a short generation interval, which shortens the time for testing the effects of the introduced mutation several fold.…”
Section: Gene Editingmentioning
confidence: 99%
“…In the case of erythroid disorders, previous studies have identified GATA1 binding site mutations located in noncoding regions of a number of genes implicated in human erythroid disorders: ALAS2 in X-linked sideroblastic anemia (25,26), PKLR in pyruvate kinase deficiency (27), and UROS in congenital erythropoietic porphyria (28). These noncoding variants provide a unique opportunity to examine the necessity of these elements using modern genome editing tools, such as CRISPR/Cas9 (29). It is likely that such genome editing approaches coupled with human genetic analysis of wholegenome sequencing data, will help to identify additional noncoding variants implicated in human disease.…”
Section: Insight From Rare Disorders Of Erythropoiesismentioning
confidence: 99%