caa a22 4500 606190376 CHVBK 20210128100855.0 cr unu---uuuuu 210128e20150201xx s 000 0 eng 10.1007/s10577-014-9459-z doi (NATIONALLICENCE)springer-10.1007/s10577-014-9459-z A pathway from chromosome transfer to engineering resulting in human and mouse artificial chromosomes for a variety of applications to bio-medical challenges [Elektronische Daten] [Mitsuo Oshimura, Narumi Uno, Yasuhiro Kazuki, Motonobu Katoh, Toshiaki Inoue] Microcell-mediated chromosome transfer (MMCT) is a technique to transfer a chromosome from defined donor cells into recipient cells and to manipulate chromosomes as gene delivery vectors and open a new avenue in somatic cell genetics. However, it is difficult to uncover the function of a single specific gene via the transfer of an entire chromosome or fragment, because each chromosome or fragment contains a set of numerous genes. Thus, alternative tools are human artificial chromosome (HAC) and mouse artificial chromosome (MAC) vectors, which can carry a gene or genes of interest. HACs/MACs have been generated mainly by either a "top-down approach” (engineered creation) or a "bottom-up approach” (de novo creation). HACs/MACs with one or more acceptor sites exhibit several characteristics required by an ideal gene delivery vector, including stable episomal maintenance and the capacity to carry large genomic loci plus their regulatory elements, thus allowing the physiological regulation of the introduced gene in a manner similar to that of native chromosomes. The MMCT technique is also applied for manipulating HACs and MACs in donor cells and delivering them to recipient cells. This review describes the lessons learned and prospects identified from studies on the construction of HACs and MACs, and their ability to drive exogenous gene expression in cultured cells and transgenic animals via MMCT. New avenues for a variety of applications to bio-medical challenges are also proposed. The Author(s), 2015 Microcell-mediated chromosome transfer nationallicence Human artificial chromosome nationallicence Mouse artificial chromosome nationallicence Chromosome engineering nationallicence Humanized model mouse nationallicence Gene-/cell-therapy nationallicence 6-TG : 6-thioguanine nationallicence BAC : Bacterial artificial chromosome nationallicence BRCA-1 : Breast cancer susceptibility gene I nationallicence BRMS1 : Breast cancer metastasis-suppressor 1 nationallicence CENP-A : Centromere protein A nationallicence CHO : Chinese hamster ovary nationallicence CRISPR : Clustered regulatory interspaced short palindromic repeat nationallicence CSN2 : Beta-casein nationallicence CYP3A : Cytochrome P450, family 3, subfamily A nationallicence CYP3A-HAC : CYP3A cluster-containing HAC nationallicence DMD : Duchenne musclular dystrophy nationallicence DS : Down syndrome nationallicence DYS-HAC : Genomic dystrophin locus-containing HAC nationallicence EGFP : Enhanced green fluorescent protein nationallicence ES : Embryonic stem nationallicence FVIII : Human factor VIII nationallicence GALC : Galactosylceramidase nationallicence GATA1s : GATA-binding protein 1 shorter isoform nationallicence GCH1 : GTP cyclohydrolase 1 nationallicence H/F protein : Hemagglutinin/fusion protein nationallicence HAC : Human artificial chromosome nationallicence HAT : Hypoxanthine-aminopterin-thymidine nationallicence hCFs : Human chromosome fragments nationallicence hiMSC : Human immortalized mesenchymal stem cell nationallicence HLA : Human Leukocyte Antigen nationallicence HPRT : Hypoxanthine phosphoribosyl transferase nationallicence iHAC : iPS cell-inducible HAC nationallicence iPS : Induced pluripotent stem nationallicence LIT1(KCNQ1OT1) : Imprinted antisense RNA in the human KvLQT1 nationallicence MAC : Mouse artificial chromosome nationallicence MEFs : Mouse embryonic fibroblasts nationallicence mGS cells : Multipotent germline stem cells nationallicence MI-HAC : Multi-integrase HAC nationallicence MMCT : Microcell-mediated chromosome transfer nationallicence MUC4 : Mucin 4 nationallicence MV : Measles virus nationallicence NBS1 : Nijmegen breakage syndrome 1 nationallicence OPN : Osteopontin nationallicence PAC : P1-derived artificial chromosome nationallicence PDLs : Population doubling levels nationallicence PEG : Polyethylene glycol nationallicence PITX1 : Paired-like homeodomain 1 nationallicence SATAC : Satellite-DNA-based artificial chromosome nationallicence scFv : Single-chain variable fragment nationallicence SIM : Simultaneous or sequential integration of multiple gene-loading vectors nationallicence ssODN : Single-strand donor oligonucleotides nationallicence TALE : Transactivator like effector nationallicence TALENs : Transactivator like effector nucleases nationallicence TAM : Transient abnormal myelopoiesis nationallicence TAR : Transformation-associated recombination nationallicence Tc mouse : Trans-chromosomic mouse nationallicence tet-O : Tetracycline operator nationallicence TfR : Transferrin receptor nationallicence Ts21 : Trisomy 21 nationallicence VHL : Von Hippel-Lindau tumor suppressor nationallicence YAC : Yeast artificial chromosome nationallicence ZFP : Zinc finger protein nationallicence ZFNs : Zinc finger nucleases nationallicence Oshimura Mitsuo Chromosome Engineering Research Center, Tottori University, 86 Nishi-cho, 683-8503, Yonago, Tottori, Japan aut Uno Narumi Chromosome Engineering Research Center, Tottori University, 86 Nishi-cho, 683-8503, Yonago, Tottori, Japan aut Kazuki Yasuhiro Chromosome Engineering Research Center, Tottori University, 86 Nishi-cho, 683-8503, Yonago, Tottori, Japan aut Katoh Motonobu Division of Human Genome Science, School of Life Sciences, Faculty of Medicine, Tottori University, 86 Nishi-cho, 683-8503, Yonago, Tottori, Japan aut Inoue Toshiaki Division of Human Genome Science, School of Life Sciences, Faculty of Medicine, Tottori University, 86 Nishi-cho, 683-8503, Yonago, Tottori, Japan aut Chromosome Research Springer Netherlands 23/1(2015-02-01), 111-133 0967-3849 23:1<111 2015 23 10577 https://doi.org/10.1007/s10577-014-9459-z text/html Onlinezugriff via DOI BK010053 XK010053 XK010000 Metadata rights reserved Springer special CC-BY-NC licence nationallicence 1 research-article jats NATIONALLICENCE NATIONALLICENCE NL-springer NATIONALLICENCE 856 40 https://doi.org/10.1007/s10577-014-9459-z text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Oshimura Mitsuo Chromosome Engineering Research Center, Tottori University, 86 Nishi-cho, 683-8503, Yonago, Tottori, Japan aut NATIONALLICENCE 700 1- Uno Narumi Chromosome Engineering Research Center, Tottori University, 86 Nishi-cho, 683-8503, Yonago, Tottori, Japan aut NATIONALLICENCE 700 1- Kazuki Yasuhiro Chromosome Engineering Research Center, Tottori University, 86 Nishi-cho, 683-8503, Yonago, Tottori, Japan aut NATIONALLICENCE 700 1- Katoh Motonobu Division of Human Genome Science, School of Life Sciences, Faculty of Medicine, Tottori University, 86 Nishi-cho, 683-8503, Yonago, Tottori, Japan aut NATIONALLICENCE 700 1- Inoue Toshiaki Division of Human Genome Science, School of Life Sciences, Faculty of Medicine, Tottori University, 86 Nishi-cho, 683-8503, Yonago, Tottori, Japan aut NATIONALLICENCE 773 0- Chromosome Research Springer Netherlands 23/1(2015-02-01), 111-133 0967-3849 23:1<111 2015 23 10577