title: Biological and biomedical applications of engineered nucleases.
abstract: The development of engineered nucleases is the fruit of a new technological approach developed in the last two decades which has led to significant benefits on genome engineering, particularly on gene therapy. These applications enable efficient and specific genetic modifications via the induction of a double-strand break (DSB) in a specific genomic target sequence, followed by the homology-directed repair (HDR) or non-homologous end joining (NHEJ) pathways. In addition to the application on gene modification in cells and intact organisms, a number of recent papers have reported that this gene editing technology can be applied effectively to human diseases. With the promising data obtained using engineered endonucleases in gene therapy, it appears reasonable to expect that more diseases could be treated and even be cured in this new era of individualized medicine. This paper first brief introduces the development of engineered nucleases with a special emphasis on zinc-finger nucleases (ZFNs) and transcription activator-like effector (TALE) nucleases (TALENs), and then takes CCR5-based gene therapy as an example to discuss the therapeutic applications of engineered nucleases.
authors.affiliation: Department of Molecular Diagnostics and Biopharmaceutics, College of Pharmaceutical Science, Soochow University, Suzhou 215123, China.
authors.forename: Yunzhi
authors.initials: Y
authors.lastname: Pan
authors.forename: Li
authors.initials: L
authors.lastname: Xiao
authors.forename: Alice S S
authors.initials: AS
authors.lastname: Li
authors.forename: Xu
authors.initials: X
authors.lastname: Zhang
authors.forename: Pierre
authors.initials: P
authors.lastname: Sirois
authors.forename: Jia
authors.initials: J
authors.lastname: Zhang
authors.forename: Kai
authors.initials: K
authors.lastname: Li
mesh_terms: D065100:CCR5 Receptor Antagonists; D053903:DNA Breaks, Double-Stranded; D059766:DNA End-Joining Repair; D004720:Endonucleases / Q000235:genetics* / Q000627:therapeutic use; D015316:Genetic Therapy / Q000379:methods; D015894:Genome, Human; D006678:HIV / Q000187:drug effects; D015658:HIV Infections / Q000235:genetics / Q000628:therapy*; D006801:Humans; D016254:Mutagenesis, Insertional; D015202:Protein Engineering; D019713:Receptors, CCR5 / Q000235:genetics; D059767:Recombinational DNA Repair; D017384:Sequence Deletion; D016335:Zinc Fingers / Q000235:genetics*
chemical_list: D065100:CCR5 Receptor Antagonists; C584933:CCR5 protein, human; D019713:Receptors, CCR5; D004720:Endonucleases