RETROVIRAL-MEDIATED GENE-THERAPY FOR THE TREATMENT OF CITRULLINEMIA - TRANSFER AND EXPRESSION OF ARGININOSUCCINATE SYNTHETASE IN HUMAN HEMATOPOIETIC-CELLS

被引：3
作者：
DEMARQUOY, J [1 ]
机构：
[1] BAYLOR COLL MED,INST MOLEC GENET,HOUSTON,TX 77030
来源：
EXPERIENTIA | 1993年 / 49卷 / 04期
关键词：
GENE THERAPY; RETROVIRUS; CITRULLINEMIA; HUMAN;
D O I：
10.1007/BF01923419
中图分类号：
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号：
07 ; 0710 ; 09 ;
摘要：
Citrullinemia is a recessive genetic disease caused by a deficiency in argininosuccinate synthetase (AS). Retroviruses were used to transduce the human AS gene into cultured human cells. Using amphotropic viruses with high titer (> 10(6) cfu/ml), we were able to correct the defect in cultured fibroblasts from citrullinemic patients. Retroviral transduction of the human AS gene into human bone marrow cells was also studied. Co-cultivation was used to infect the cells and up to 80% of progenitor cells were found to be carrying and expressing the AS retrovirus after infection. When the infected cells were kept in culture, integration and expression of the retrovirus was observed. Retroviral sequences were present and expressed in the cultured bone marrow-derived cells for up to 10 weeks.
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页码：345 / 348
页数：4
相关论文
共 22 条
[1]  
BEAUDET AL, 1986, ADV HUM GENET, V15, P161
[2]   SEQUENCE FOR HUMAN ARGININOSUCCINATE SYNTHETASE CDNA [J].
BOCK, HGO ;
SU, TS ;
OBRIEN, WE ;
BEAUDET, AL .
NUCLEIC ACIDS RESEARCH, 1983, 11 (18) :6505-6512
[3]  
Brusilow S.W., 1989, METABOLIC BASIS INHE, P629
[4]   GENE-THERAPY - A NEW APPROACH FOR THE TREATMENT OF GENETIC-DISORDERS [J].
COURNOYER, D ;
SCARPA, M ;
JONES, SN ;
MOORE, KA ;
BELMONT, JW ;
CASKEY, CT .
CLINICAL PHARMACOLOGY & THERAPEUTICS, 1990, 47 (01) :1-11
[5]   GENE-TRANSFER OF ADENOSINE-DEAMINASE INTO PRIMITIVE HUMAN HEMATOPOIETIC PROGENITOR CELLS [J].
COURNOYER, D ;
SCARPA, M ;
MITANI, K ;
MOORE, KA ;
MARKOWITZ, D ;
BANK, A ;
BELMONT, JW ;
CASKEY, CT .
HUMAN GENE THERAPY, 1991, 2 (03) :203-213
[6]   LONG-TERM EXPRESSION OF HUMAN ARGININOSUCCINATE SYNTHETASE IN MICE FOLLOWING BONE-MARROW TRANSPLANTATION WITH RETROVIRUS-TRANSDUCED HEMATOPOIETIC STEM-CELLS [J].
DEMARQUOY, J ;
HERMAN, GE ;
LORENZO, I ;
TRENTIN, J ;
BEAUDET, AL ;
OBRIEN, WE .
HUMAN GENE THERAPY, 1992, 3 (01) :3-10
[7]  
DEMARQUOY J, 1992, CLIN BIOTECHNOL, V3, P209
[8]   MOLECULAR DEFINITION OF BOVINE ARGININOSUCCINATE SYNTHETASE DEFICIENCY [J].
DENNIS, JA ;
HEALY, PJ ;
BEAUDET, AL ;
OBRIEN, WE .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1989, 86 (20) :7947-7951
[9]   CONDITIONS CONTROLLING PROLIFERATION OF HEMATOPOIETIC STEM-CELLS INVITRO [J].
DEXTER, TM ;
ALLEN, TD ;
LAJTHA, LG .
JOURNAL OF CELLULAR PHYSIOLOGY, 1977, 91 (03) :335-344
[10]   MOLECULAR-STRUCTURE OF THE HUMAN ARGININOSUCCINATE SYNTHETASE GENE - OCCURRENCE OF ALTERNATIVE MESSENGER-RNA SPLICING [J].
FREYTAG, SO ;
BEAUDET, AL ;
BOCK, HGO ;
OBRIEN, WE .
MOLECULAR AND CELLULAR BIOLOGY, 1984, 4 (10) :1978-1984
123