Efficient genomic correction methods in human iPS cells using CRISPR–Cas9 system

Li, Hongmei Lisa; Gee, Peter; Ishida, Kentaro; Hotta, Akitsu

doi:10.1016/j.ymeth.2015.10.015

Cited by 61 publications

(47 citation statements)

References 64 publications

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“…The CRISPR-Cas9 system could treat this illness by excluding the trinucleotide repetition from the patient's DNA. By bringing together the CRISPR-Cas9 system and induced pluripotent stem cell (IPSC) the editing process could be improved [24], and there are several strategies for this procedure [25]. Besides Huntington's disease, this technique could be performed in Rett syndrome [26] and schizophrenia [27].…”

Section: Medicinementioning

confidence: 99%

Review. Development, Applications, Benefits, Challenges and Limitations of the New Genome Engineering Technique. An Update Study

Crauciuc

Tripon

Gheorghiu

et al. 2017

Acta Medica Marisiensis

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We assume that the CRISPR Cas9 theory must be delimited by applicability, because the consequences of long term DNA manipulation remain unknown. Moreover, the irreversibility of this procedure should instigate researchers to reserved opinions. Usefulness as well as benefits of CRISPR Cas9 made it one of the most popular and used genome editing technique. But with its huge potential, ethical and safety concerns emerge. Therefore, before continuing research in this direction we should have a well organized system that is able to make that differentiation between research and reproduction. However we truly believe in the future of genetic engineering and with the CRISPR-Cas9 system we expect that the opportunity of treating now so called incurable diseases arises. Time is all we need.

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Section: Medicinementioning

confidence: 99%

Review. Development, Applications, Benefits, Challenges and Limitations of the New Genome Engineering Technique. An Update Study

Crauciuc

Tripon

Gheorghiu

et al. 2017

Acta Medica Marisiensis

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show abstract

“…1e) [48,71]. Generally, the distance of SNV from the DSB should be minimized for efficient SNV incorporation [72,73]. This method is routinely performed in early embryos of many animal species [74][75][76].…”

Section: Ssodn-mediated Single Nucleotide Variation (Snv) Knock-inmentioning

confidence: 99%

Updated summary of genome editing technology in human cultured cells linked to human genetics studies

2017

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Current deep-sequencing technology provides a mass of nucleotide variations associated with human genetic disorders to accelerate the identification of causative mutations. To understand the etiology of genetic disorders, reverse genetics in human cultured cells is a useful approach for modeling a disease in vitro. However, gene targeting in human cultured cells is difficult because of their low activity of homologous recombination. Engineered endonucleases enable enhancement of the local activation of DNA repair pathways at the human genome target site to rewrite the desired sequence, thereby efficiently generating disease-modeling cultured cell clones. These edited cells can be used to explore the molecular functions of a causative gene product to uncover the etiological mechanisms. The correction of mutations in patient cells using genome editing technology could contribute to the development of unique gene therapies. This technology can also be applied to screening causative mutations. Rare genetic disorders and non-exonic mutation-caused diseases remain frontier in the field of human genetics as it is difficult to validate whether the extracted nucleotide variants are mutation or polymorphism. When isogenic human cultured cells with a candidate variant reproduce the pathogenic phenotypes, it is confirmed that the variant is a causative mutation.

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“…3). CRISPR/Cas9 is an efficient method for both constructing disease models in wild-type iPSCs [16] and for correcting disease genes in patient-derived iPSCs [18].…”

Section: Application Of Crispr/cas9mentioning

confidence: 99%

Genome editing: A breakthrough in life science and medicine [Review]

Hatada

Horii

2016

Endocr J

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Efficient genomic correction methods in human iPS cells using CRISPR–Cas9 system

Cited by 61 publications

References 64 publications

Review. Development, Applications, Benefits, Challenges and Limitations of the New Genome Engineering Technique. An Update Study

Review. Development, Applications, Benefits, Challenges and Limitations of the New Genome Engineering Technique. An Update Study

Updated summary of genome editing technology in human cultured cells linked to human genetics studies

Genome editing: A breakthrough in life science and medicine [Review]

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