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

被引：7

作者：

Katona, R. L. ^{[1
]}

Sinko, I. ^{[1
]}

Hollo, G. ^{[1
]}

Szuecs, K. Szekely ^{[1
]}

Praznovszky, T. ^{[1
]}

Keresoe, J. ^{[1
]}

Csonka, E. ^{[1
]}

Fodor, K. ^{[1
]}

Cserpan, I. ^{[1
]}

Szakal, B. ^{[1
]}

Blazso, P. ^{[1
]}

Udvardy, A. ^{[2
]}

Hadlaczky, G. ^{[1
]}

机构：

[1] Hungarian Acad Sci, Inst Genet, Biol Res Ctr, H-6726 Szeged, Hungary

[2] Hungarian Acad Sci, Inst Biochem, Biol Res Ctr, H-6726 Szeged, Hungary

来源：

CELLULAR AND MOLECULAR LIFE SCIENCES | 2008年 / 65卷 / 23期

关键词：

Mammalian artificial chromosome; stem cell; Krabbe's disease; transgenic mouse; gene therapy;

D O I：

10.1007/s00018-008-8442-2

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：

Mammalian artificial chromosomes (MACs) are safe, stable, non-integrating genetic vectors with almost unlimited therapeutic transgene-carrying capacity. The combination of MAC and stem cell technologies offers a new strategy for stem cell-based therapy, the efficacy of which was confirmed and validated by using a mouse model of a devastating monogenic disease, galactocerebrosidase deficiency (Krabbe's disease). Therapeutic MACs were generated by sequence-specific loading of galactocerebrosidase transgenes into a platform MAC, and stable, pluripotent mouse embryonic stem cell lines were established with these chromosomes. The transgenic stem cells were thoroughly characterized and used to produce chimeric mice on the mutant genetic background. The lifespan of these chimeras was increased twofold, verifying the feasibility of the development of MAC-stem cell systems for the delivery of therapeutic genes in stem cells to treat genetic diseases and cancers, and to produce cell types for cell replacement therapies.

引用

页码：3830 / 3838

页数：9

共 37 条

[1] EMBRYONIC STEM-CELLS EXPRESS NEURONAL PROPERTIES IN-VITRO [J].

BAIN, G ;

KITCHENS, D ;

YAO, M ;

HUETTNER, JE ;

GOTTLIEB, DI .

DEVELOPMENTAL BIOLOGY, 1995, 168 (02) :342-357

[2] EXTENT OF SEQUENCE HOMOLOGY REQUIRED FOR BACTERIOPHAGE-LAMBDA SITE-SPECIFIC RECOMBINATION [J].

BAUER, CE ;

GARDNER, JF ;

GUMPORT, RI .

JOURNAL OF MOLECULAR BIOLOGY, 1985, 181 (02) :187-197

[3] Delayed clinical and pathological signs in twitcher (Globoid cell leukodystrophy) mice on a C57BL/6 x CAST/Ei background [J].

Biswas, S ;

Biesiada, H ;

Williams, TD ;

LeVine, SM .

NEUROBIOLOGY OF DISEASE, 2002, 10 (03) :344-357

[4] Reciprocal transcriptional regulation of Pou5f1 and Sox2 via the Oct4/Sox2 complex in embryonic stem cells [J].

Chew, JL ;

Loh, YH ;

Zhang, WS ;

Chen, X ;

Tam, WL ;

Yeap, LS ;

Li, P ;

Ang, YS ;

Lim, B ;

Robson, P ;

Ng, HH .

MOLECULAR AND CELLULAR BIOLOGY, 2005, 25 (14) :6031-6046

[5] Characterization of the chicken beta-globin insulator [J].

Chung, JH ;

Bell, AC ;

Felsenfeld, G .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1997, 94 (02) :575-580

[6] Transgenic rescue of Krabbe disease in the twitcher mouse [J].

De Gasperi, R ;

Friedrich, VL ;

Perez, GM ;

Senturk, E ;

Wen, PH ;

Kelley, K ;

Elder, GA ;

Sosa, MAG .

GENE THERAPY, 2004, 11 (15) :1188-1194

[7] Efficient in-vitro transfer of a 60-Mb mammalian artificial chromosome into murine and hamster cells using cationic lipids and dendrimers [J].

de Jong, G ;

Telenius, A ;

Vanderbyl, S ;

Meitz, A ;

Drayer, J .

CHROMOSOME RESEARCH, 2001, 9 (06) :475-485

[8] Design and optimization of lentiviral vectors for transfer of GALC expression in Twitcher brain [J].

Dolcetta, D. ;

Perani, L. ;

Givogri, M. I. ;

Galbiati, F. ;

Amadio, S. ;

Del Carro, U. ;

Finocchiaro, G. ;

Fanzani, A. ;

Marchesini, S. ;

Naldini, L. ;

Roncarolo, M. G. ;

Bongarzone, E. .

JOURNAL OF GENE MEDICINE, 2006, 8 (08) :962-971

[9] Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease [J].

Escolar, ML ;

Poe, MD ;

Provenzale, JM ;

Richards, KC ;

Allison, J ;

Wood, S ;

Wenger, DA ;

Pietryga, D ;

Wall, D ;

Champagne, M ;

Morse, R ;

Krivit, W ;

Kurtzberg, J .

NEW ENGLAND JOURNAL OF MEDICINE, 2005, 352 (20) :2069-2081

[10] CENTROMERE PROTEINS .1. MITOSIS SPECIFIC CENTROMERE ANTIGEN RECOGNIZED BY ANTI-CENTROMERE AUTOANTIBODIES [J].

HADLACZKY, G ;

PRAZNOVSZKY, T ;

RASKO, I ;

KERESO, J .

CHROMOSOMA, 1989, 97 (04) :282-288

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