title: Applications of TALENs and CRISPR/Cas9 in human cells and their potentials for gene therapy.
abstract: The newly developed TALENs and emerging CRISPR/Cas9 have spurred interests in the field of genome engineering because of their ease of customization and high-efficient site-specific cleavages. Although these novel technologies have been successfully used in many types of cells, it is of great importance to apply them in human-derived cells to further observe and evaluate their clinical potentials in gene therapy. Here, we review the working mechanism of TALEN and CRISPR/Cas9, their effectiveness and specificity in human cells, and current methods to enhance efficiency and reduce off-target effects. Besides, CCR5 gene was chosen as a target example to illustrate their clinical potentials. Finally, some questions are raised for future research and for researchers to consider when making a proper choice bases on different purposes.
authors.affiliation: Department of Hematopoietic Stem Cell Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, No. 8 Dongda Street, Fengtai District, Beijing, 100071, People's Republic of China.
authors.forename: Jingwen
authors.initials: J
authors.lastname: Niu
authors.forename: Bin
authors.initials: B
authors.lastname: Zhang
authors.forename: Hu
authors.initials: H
authors.lastname: Chen
mesh_terms: D001709:Biotechnology; D064113:CRISPR-Cas Systems; D004260:DNA Repair; D018390:Gene Targeting; D005818:Genetic Engineering / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D008957:Models, Genetic; D017393:RNA Editing; D019713:Receptors, CCR5 / Q000235:genetics
chemical_list: C584933:CCR5 protein, human; D019713:Receptors, CCR5