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Recent developments and clinical studies utilizing engineered zinc finger nuclease technology

DC FieldValueLanguage
dc.contributor.author김형범-
dc.date.accessioned2016-02-04T11:54:41Z-
dc.date.available2016-02-04T11:54:41Z-
dc.date.issued2015-
dc.identifier.issn1420-682X-
dc.identifier.urihttps://ir.ymlib.yonsei.ac.kr/handle/22282913/141471-
dc.description.abstractEfficient methods for creating targeted genetic modifications have long been sought for the investigation of gene function and the development of therapeutic modalities for various diseases, including genetic disorders. Although such modifications are possible using homologous recombination, the efficiency is extremely low. Zinc finger nucleases (ZFNs) are custom-designed artificial nucleases that make double-strand breaks at specific sequences, enabling efficient targeted genetic modifications such as corrections, additions, gene knockouts and structural variations. ZFNs are composed of two domains: (i) a DNA-binding domain comprised of zinc finger modules and (ii) the FokI nuclease domain that cleaves the DNA strand. Over 17 years after ZFNs were initially developed, a number of improvements have been made. Here, we will review the developments and future perspectives of ZFN technology. For example, ZFN activity and specificity have been significantly enhanced by modifying the DNA-binding domain and FokI cleavage domain. Advances in culture methods, such as the application of a cold shock and the use of small molecules that affect ZFN stability, have also increased ZFN activity. Furthermore, ZFN-induced mutant cells can be enriched using episomal surrogate reporters. Additionally, we discuss several ongoing clinical studies that are based on ZFN-mediated genome editing in humans. These breakthroughs have substantially facilitated the use of ZFNs in research, medicine and biotechnology.-
dc.description.statementOfResponsibilityopen-
dc.relation.isPartOfCellular and Molecular Life Sciences-
dc.rightsCC BY-NC-ND 2.0 KR-
dc.rights.urihttps://creativecommons.org/licenses/by-nc-nd/2.0/kr/-
dc.subject.MESHAnimals-
dc.subject.MESHAnimals, Domestic/genetics-
dc.subject.MESHCell Culture Techniques-
dc.subject.MESHDeoxyribonucleases/chemistry*-
dc.subject.MESHGenetic Engineering/methods*-
dc.subject.MESHGenetic Therapy/methods-
dc.subject.MESHGenetic Therapy/trends-
dc.subject.MESHZinc Fingers*-
dc.titleRecent developments and clinical studies utilizing engineered zinc finger nuclease technology-
dc.typeArticle-
dc.contributor.collegeCollege of Medicine (의과대학)-
dc.contributor.departmentDept. of Pharmacology (약리학)-
dc.contributor.googleauthorYoung-Il Jo-
dc.contributor.googleauthorHyongbum Kim-
dc.contributor.googleauthorSuresh Ramakrishna-
dc.identifier.doi10.1007/s00018-015-1956-5.-
dc.admin.authorfalse-
dc.admin.mappingfalse-
dc.contributor.localIdA01148-
dc.relation.journalcodeJ00496-
dc.identifier.pmid26089249-
dc.identifier.urlhttp://link.springer.com/article/10.1007%2Fs00018-015-1956-5-
dc.subject.keywordFarm animals-
dc.subject.keywordPre-clinical trials-
dc.subject.keywordProgrammable nucleases-
dc.subject.keywordTargeted genetic modifications-
dc.subject.keywordTherapeutic applications-
dc.subject.keywordZFN architecture-
dc.subject.keywordZFN delivery-
dc.subject.keywordZFN modification-
dc.contributor.alternativeNameKim, Hyongbum-
dc.contributor.affiliatedAuthorKim, Hyongbum-
dc.rights.accessRightsnot free-
dc.citation.volume72-
dc.citation.number20-
dc.citation.startPage3819-
dc.citation.endPage3830-
dc.identifier.bibliographicCitationCellular and Molecular Life Sciences, Vol.72(20) : 3819-3830, 2015-
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pharmacology (약리학교실) > 1. Journal Papers

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