Progress and prospects: human artificial chromosomes

被引：21

作者：

Macnab, S. ^{[1
,2
]}

Whitehouse, A. ^{[1
,2
]}

机构：

[1] Univ Leeds, Fac Biol Sci, Inst Mol & Cellular Biol, Leeds LS2 9JT, W Yorkshire, England

[2] Univ Leeds, Astbury Ctr Struct Mol Biol, Leeds LS2 9JT, W Yorkshire, England

来源：

GENE THERAPY | 2009年 / 16卷 / 10期

关键词：

HACs; BACs; YACs; amplicons; genomic transgenes; stem cells; STEM-CELLS; DNA; DELIVERY; EXPRESSION; GENOMES; CLONING; BAC;

D O I：

10.1038/gt.2009.102

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Artificial chromosomes (ACs) are highly promising vectors for use in gene therapy applications. They are able to maintain expression of genomic-sized exogenous transgenes within target cells, without integrating into the host genome. Although these vectors have huge potential and benefits when compared against normal expression constructs, they are highly complex, technically challenging to construct and diffcult to deliver to target cells. This review focuses on the current progress in the field of ACs and discusses the recent advances in purification, construction, delivery and potential new molecular therapies. Gene Therapy (2009) 16, 1180-1188; doi: 10.1038/gt.2009.102; published online 27 August 2009

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收藏

页码：1180 / 1188

页数：9

相关论文

共 27 条

[1] Chromosomal engineering [J].

Duncan, Alistair ;

Hadlaczky, Gyula .

CURRENT OPINION IN BIOTECHNOLOGY, 2007, 18 (05) :420-424

[2] Infectious delivery and expression of a 135 kb human FRDA genomic DNA locus complements Friedreich's ataxia deficiency in human cells [J].

Gomez-Sebastian, Silvia ;

Gimenez-Cassina, Alfredo ;

Diaz-Nido, Javier ;

Lim, Filip ;

Wade-Martins, Richard .

MOLECULAR THERAPY, 2007, 15 (02) :248-254

[3] Engineering Cell Lines for Production of Replication Defective HSV-1 Gene Therapy Vectors [J].

Grant, Kyle G. ;

Krisky, David M. ;

Ataai, Mohammed M. ;

Glorioso, Joseph C., III .

BIOTECHNOLOGY AND BIOENGINEERING, 2009, 102 (04) :1087-1097

[4] Herpesvirus saimiri-based gene delivery vectors [J].

Griffiths, RA ;

Boyne, JA ;

Whitehouse, A .

CURRENT GENE THERAPY, 2006, 6 (01) :1-15

[5] Artificial chromosome-based transgenes in the study of genome function [J].

Heaney, Jason D. ;

Bronson, Sarah K. .

MAMMALIAN GENOME, 2006, 17 (08) :791-807

[6] Delivery and long-term expression of a 135 kb LDLR genomic DNA locus in vivo by hydrodynamic tail vein injection [J].

Hibbitt, Olivia C. ;

Harbottle, Richard P. ;

Waddington, Simon N. ;

Bursill, Christine A. ;

Coutelle, Charles ;

Keith, Channoni ;

Wade-Martins, Richard .

JOURNAL OF GENE MEDICINE, 2007, 9 (06) :488-497

[7] Delivery of large genomic DNA inserts >100 kb using HSV-1 amplicons [J].

Hibbitt, Olivia C. ;

Wade-Martins, Richard .

CURRENT GENE THERAPY, 2006, 6 (03) :325-336

[8] A highly Stable and Nonintegrated Human Artificial Chromosome (HAC) Containing the 2.4 Mb Entire Human Dystrophin Gene [J].

Hoshiya, Hidetoshi ;

Kazuki, Yasuhiro ;

Abe, Satoshi ;

Takiguchi, Masato ;

Kajitani, Naoyo ;

Watanabe, Yoshinori ;

Yoshino, Toko ;

Shirayoshi, Yasuaki ;

Higaki, Katsumi ;

Messina, Graziella ;

Cossu, Giulio ;

Oshimura, Mitsuo .

MOLECULAR THERAPY, 2009, 17 (02) :309-317

[9] E2F-dependent repression of topoisomerase II regulates heterochromatin formation and apoptosis in cells with melanoma-prone mutation [J].

Jiao, W ;

Lin, HN ;

Timmons, J ;

Nagaich, AK ;

Ng, SW ;

Misteli, T ;

Rane, SG .

CANCER RESEARCH, 2005, 65 (10) :4067-4077

[10] A combined artificial chromosome-stem cell therapy method in a model experiment aimed at the treatment of Krabbe's disease in the Twitcher mouse [J].

Katona, R. L. ;

Sinko, I. ;

Hollo, G. ;

Szuecs, K. Szekely ;

Praznovszky, T. ;

Keresoe, J. ;

Csonka, E. ;

Fodor, K. ;

Cserpan, I. ;

Szakal, B. ;

Blazso, P. ;

Udvardy, A. ;

Hadlaczky, G. .

CELLULAR AND MOLECULAR LIFE SCIENCES, 2008, 65 (23) :3830-3838