Technology developments in biological tools for targeted genome surgery

Teimourian, Shahram; Abdollahzadeh, Rasoul

doi:10.1007/s10529-014-1656-5

Cited by 8 publications

(4 citation statements)

References 67 publications

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“…To avoid or prevent significant risks of off-target mutations, we chose TALENs to achieve mutagenesis instead of CRISPR. Even though CRISPR has emerged recently as a potentially facile and efficient alternative to ZFNs and TALENs to induce targeted genetic alterations, CRISPR does not have better specificity than TALENs or ZFNs [ 17 , 26 ]. Meanwhile previous studies have shown that low efficacy and minor off-target mutations may exist in TALEN-induced animals [ 22 , 23 , 28 – 31 ].…”

Section: Discussionmentioning

confidence: 99%

“…Meanwhile previous studies have shown that low efficacy and minor off-target mutations may exist in TALEN-induced animals [ 22 , 23 , 28 – 31 ]. However, TALENs still provides a valuable option, thanks to its relatively high specificity [ 26 ]. Off-target analysis of the cloned goats revealed that no off-target sequences were detected.…”

Section: Discussionmentioning

confidence: 99%

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Efficient TALEN-mediated myostatin gene editing in goats

Lü

Yuan

et al. 2016

BMC Dev Biol

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Background: Myostatin (MSTN) encodes a negative regulator of skeletal muscle mass that might have applications for promoting muscle growth in livestock. In this study, we aimed to test whether targeted MSTN editing, mediated by transcription activator-like effector nucleases (TALENs), is a viable approach to create myostatin-modified goats (Capra hircus). Results: We obtained a pair of TALENs (MTAL-2) that could recognize and cut the targeted MSTN site in the goat genome. Fibroblasts from pedigreed goats were co-transfected with MTAL-2, and 272 monoclonal cell strains were confirmed to have mono-or bi-allelic mutations in MSTN. Ten cell strains with different genotypes were used as donor cells for somatic cell nuclear transfer, which produced three cloned kids (K179/MSTN −/− , K52-2/MSTN +/− , and K52-1/MSTN +/+ ).

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Efficient TALEN-mediated myostatin gene editing in goats

Lü

Yuan

et al. 2016

BMC Dev Biol

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“…Other advantages of using phiC31 integrase include non-viral delivery, sustainable transgene expression, and functions in species like bacteria, yeast, plants, frogs, chickens, mice, rats, pigs, cows, and humans. [2,73] However, potential sites of phiC31-based genome editing are limited. In human genome, of the 106 mapped integration sites of phiC31, ~ 39% are within coding genes and ~ 61% are in the intergenic regions.…”

Section: Meganucleasementioning

confidence: 99%

Application of Genome Editing Technology to MicroRNA Research in Mammalians

Lei¹,

Batara²,

Lü³

2016

Modern Tools for Genetic Engineering

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Targeted nucleases have recently emerged as a powerful genome editing tool. The ability of introducing targeted, desired changes into mammalian genome makes them an invaluable tool to unravel functions of miRN"s in biology and disease. In combination with homologous donor vector, targeted nucleases can achieve high efficiency and precision, enabling bi-allelic ablation of miRN" in cultured somatic cells. Here we review the structure and function of miRN" as well as the unique implementation of genome editing technology in modifying miRN" sequences in mammalians. This chapter discusses the four mainstay genome editing technologies meganuclease, zinc finger nuclease ZFN , transcription activator-like effector nuclease T"LEN and clustered regularly interspaced short palindromic repeat-associated nuclease Cas CRISPR-Cas , focusing on T"LEN.

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“…1 ). 8 , 9 , 10 The deletions or excisions of DNA induced by CRISPR in order to restore ORF is a new and encouraging approach in treating patients caused by deletion and duplication mutations that disrupt ORF. Also, the precise correction of the mutation through CRISPR-induced homologous directed repair (HDR) is another hallmark that brings by CRISPR/Cas9 technology for the treatment of DMD.…”

Section: Introductionmentioning

confidence: 99%

Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy

et al. 2021

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Duchenne muscular dystrophy is an X-linked recessive hereditary monogenic disorder caused by inability to produce dystrophin protein. In most patients, the expression of dystrophin lost due to disrupting mutations in open reading frame. Despite the efforts in a large number of different therapeutic approaches to date, the treatments available for DMD remain mitigative and supportive to improve the symptoms of the disease, rather than to be curative. The advent of CRISPR/Cas9 technology has revolutionized genome editing scope and considered as pioneer in effective genomic engineering. Deletions or excisions of intragenic DNA by CRISPR as well as a similar strategy with exon skipping at the DNA level induced by antisense oligonucleotides, are new and promising approaches in correcting DMD gene, which restore the expression of a truncated but functional dystrophin protein. Also, CRISPR/Cas9 technology can be used to treat DMD by removing duplicated exons, precise correction of causative mutation by HDR-based pathway and inducing the expression of compensatory proteins such as utrophin. In this study, we briefly explained the molecular genetics of DMD and a historical overview of DMD gene therapy. We in particular focused on CRISPR/Cas9-mediated therapeutic approaches that used to treat DMD.

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Technology developments in biological tools for targeted genome surgery

Cited by 8 publications

References 67 publications

Efficient TALEN-mediated myostatin gene editing in goats

Efficient TALEN-mediated myostatin gene editing in goats

Application of Genome Editing Technology to MicroRNA Research in Mammalians

Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy

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