Current and future delivery systems for engineered nucleases: ZFN, TALEN and RGEN

Ain, Qurrat Ul; Chung, Jee Young; Kim, Yong Hee

doi:10.1016/j.jconrel.2014.12.036

Cited by 97 publications

(52 citation statements)

References 87 publications

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“…Relatively lower expression levels of the gene ZNF 852 are predictive of seizure-free outcome following ATL/AH. Gene Ontology annotations related to this gene include nucleic acid binding [14]. Zinc finger proteins contain one or more short regions called zinc finger domains which have one of the most abundant DNA binding motifs in the eukaryotic genome recognizing any DNA sequence [14].…”

Section: Discussionmentioning

confidence: 99%

“…Gene Ontology annotations related to this gene include nucleic acid binding [14]. Zinc finger proteins contain one or more short regions called zinc finger domains which have one of the most abundant DNA binding motifs in the eukaryotic genome recognizing any DNA sequence [14]. These regions include a specific pattern of amino acids and one or more zinc ions [14].…”

Section: Discussionmentioning

confidence: 99%

“…Zinc finger proteins contain one or more short regions called zinc finger domains which have one of the most abundant DNA binding motifs in the eukaryotic genome recognizing any DNA sequence [14]. These regions include a specific pattern of amino acids and one or more zinc ions [14]. Specifically, the Cys2-His2 zinc finger domain consists of a chain of two cysteines and two histidines, which fold around a single zinc ion contacting between 3 to 18 base pairs of DNA [14].…”

Section: Discussionmentioning

confidence: 99%

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Cortical gene expression: prognostic value for seizure outcome following temporal lobectomy and amygdalohippocampectomy

et al. 2016

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Whole genome analyses were performed to test the hypothesis that temporal cortical gene expression differs between epilepsy patients rendered seizure-free versus non-seizure-free following anterior temporal lobectomy with amygdalohippocampectomy (ATL/AH). Twenty four patients underwent ATL/AH to treat medically intractable seizures of temporal lobe origin (mean age 35.5 years, mean follow up 42.2 months), they were then dichotomized into seizure-free and non-seizure-free groups. Tissue RNA was isolated from the lateral temporal cortex and gene expression analysis was performed. Whole genome data were analyzed for prognostic value for seizure-free outcome following ATL/AH by logistic regression. Genes that could distinguish seizure outcome groups were identified based on providing an accuracy of >0.90 judging by area under the receiver operating characteristic curve, AUC, with a P value of the slope coefficient of <0.05. Four genes and seven RNA probes were with prognostic value for post-operative seizure-free outcome. Gene expression associated with seizure-free outcome included relative down-regulation of: zinc finger protein 852 (ZNF852); CUB domain containing protein 2 (CDCP2); proline-rich transmembrane protein 1 (PRRT1); hypothetical LOC440200 (FLJ41170); RNA probe 8047763; RNA probe 8126238; RNA probe 8113489; RNA probe 8092883; RNA probe 7935228; RNA probe 806293 and RNA probe 8104131. This study describes the predictive value of temporal cortical gene expression for seizure-free outcome after ATL/AH. Four genes and seven RNA probes were found to predict post-operative seizure-free outcome. Future prospective investigation of these genes and probes in human brain tissue and blood could establish new biomarkers predictive of seizure outcome following ATL/AH.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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Cortical gene expression: prognostic value for seizure outcome following temporal lobectomy and amygdalohippocampectomy

et al. 2016

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“…The delivery of g-RNA and the Cas9 protein has been a constant challenge, but the researchers used plasmids, viruses, and ribonucleoproteins (RNPs) as method for optimizing their gene-editing procedures, even though these three delivery methods had limitations [44,45].…”

Section: Challenges and Limitationsmentioning

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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“…2), and articles describing successful manipulation of rodent SSCs by way of CRISPR are now available (Wu et al , 2015; Chapman et al , 2015; Sato et al , 2015). The CRISPR-Cas9 system not only bypasses the engineering of nucleases but also generates far less off-target effects compared with ZFNs (Ul Ain et al , 2015). According to a recent report, with genome-wide screens, no obvious off-target genetic or epigenetic changes could be detected in a large SSCT experiment involving CRISPR-Cas9-mediated gene targeting and transplantation of modified mouse SSCs (Wu et al , 2015).…”

Section: Clinical Prospects Of Ssct and Germline Genomic Editingmentioning

confidence: 99%

Spermatogonial stem cell autotransplantation and germline genomic editing: a future cure for spermatogenic failure and prevention of transmission of genomic diseases

Mulder

Zheng

Jan

et al. 2016

Hum. Reprod. Update

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BACKGROUNDSubfertility affects approximately 15% of all couples, and a severe male factor is identified in 17% of these couples. While the etiology of a severe male factor remains largely unknown, prior gonadotoxic treatment and genomic aberrations have been associated with this type of subfertility. Couples with a severe male factor can resort to ICSI, with either ejaculated spermatozoa (in case of oligozoospermia) or surgically retrieved testicular spermatozoa (in case of azoospermia) to generate their own biological children. Currently there is no direct treatment for azoospermia or oligozoospermia. Spermatogonial stem cell (SSC) autotransplantation (SSCT) is a promising novel clinical application currently under development to restore fertility in sterile childhood cancer survivors. Meanwhile, recent advances in genomic editing, especially the clustered regulatory interspaced short palindromic repeats-associated protein 9 (CRISPR-Cas9) system, are likely to enable genomic rectification of human SSCs in the near future.OBJECTIVE AND RATIONALEThe objective of this review is to provide insights into the prospects of the potential clinical application of SSCT with or without genomic editing to cure spermatogenic failure and to prevent transmission of genetic diseases.SEARCH METHODSWe performed a narrative review using the literature available on PubMed not restricted to any publishing year on topics of subfertility, fertility treatments, (molecular regulation of) spermatogenesis and SSCT, inherited (genetic) disorders, prenatal screening methods, genomic editing and germline editing. For germline editing, we focussed on the novel CRISPR-Cas9 system. We included papers written in English only.OUTCOMESCurrent techniques allow propagation of human SSCs in vitro, which is indispensable to successful transplantation. This technique is currently being developed in a preclinical setting for childhood cancer survivors who have stored a testis biopsy prior to cancer treatment. Similarly, SSCT could be used to restore fertility in sterile adult cancer survivors. In vitro propagation of SSCs might also be employed to enhance spermatogenesis in oligozoospermic men and in azoospermic men who still have functional SSCs albeit in insufficient numbers. The combination of SSCT with genomic editing techniques could potentially rectify defects in spermatogenesis caused by genomic mutations or, more broadly, prevent transmission of genomic diseases to the offspring. In spite of the promising prospects, SSCT and germline genomic editing are not yet clinically applicable and both techniques require optimization at various levels.WIDER IMPLICATIONSSSCT with or without genomic editing could potentially be used to restore fertility in cancer survivors to treat couples with a severe male factor and to prevent the paternal transmission of diseases. This will potentially allow these couples to have their own biological children. Technical development is progressing rapidly, and ethical reflection and societal debate on the use of SSCT with ...

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Current and future delivery systems for engineered nucleases: ZFN, TALEN and RGEN

Cited by 97 publications

References 87 publications

Cortical gene expression: prognostic value for seizure outcome following temporal lobectomy and amygdalohippocampectomy

Cortical gene expression: prognostic value for seizure outcome following temporal lobectomy and amygdalohippocampectomy

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

Spermatogonial stem cell autotransplantation and germline genomic editing: a future cure for spermatogenic failure and prevention of transmission of genomic diseases

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