期刊
NUCLEIC ACIDS RESEARCH
卷 33, 期 18, 页码 5978-5990出版社
OXFORD UNIV PRESS
DOI: 10.1093/nar/gki912
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资金
- NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES [P30AI042855] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF ENVIRONMENTAL HEALTH SCIENCES [P30ES003819] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF GENERAL MEDICAL SCIENCES [R01GM053923] Funding Source: NIH RePORTER
- NIAID NIH HHS [P30 AI042855, P30 AI42855] Funding Source: Medline
- NIEHS NIH HHS [ES 03819, P30 ES003819] Funding Source: Medline
- NIGMS NIH HHS [R01 GM053923, GM 53923] Funding Source: Medline
Custom-designed zinc finger nucleases (ZFNs), proteins designed to cut at specific DNA sequences, are becoming powerful tools in gene targeting-the process of replacing a gene within a genome by homologous recombination (HR). ZFNs that combine the non-specific cleavage domain (N) of FokI endonuclease with zinc finger proteins (ZFPs) offer a general way to deliver a site-specific double-strand break (DSB) to the genome. The development of ZFN-mediated gene targeting provides molecular biologists with the ability to site-specifically and permanently modify plant and mammalian genomes including the human genome via homology-directed repair of a targeted genomic DSB. The creation of designer ZFNs that cleave DNA at a pre-determined site depends on the reliable creation of ZFPs that can specifically recognize the chosen target site within a genome. The (Cys(2)His(2)) ZFPs offer the best framework for developing custom ZFN molecules with new sequence-specificities. Here, we explore the different approaches for generating the desired custom ZFNs with high sequence-specificity and affinity. We also discuss the potential of ZFN-mediated gene targeting for 'directed mutagenesis' and targeted 'gene editing' of the plant and mammalian genome as well as the potential of ZFN-based strategies as a form of gene therapy for human therapeutics in the future.
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