Functional correction of Fanconi anemia group C hematopoietic cells by the use of a novel lentiviral vector

被引:27
作者
Yamada, K
Olsen, JC
Patel, M
Rao, KW
Walsh, CE
机构
[1] Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA
[2] Univ N Carolina, Cyst Fibrosis Pulm Res & Treatment Ctr, Chapel Hill, NC 27599 USA
[3] Univ N Carolina, Dept Pathol, Chapel Hill, NC 27599 USA
[4] Univ N Carolina, Dept Med, Chapel Hill, NC 27599 USA
关键词
E1AV vector; lentivirus; Fanconi anemia group C (FANCC);
D O I
10.1006/mthe.2001.0287
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
Lentiviral vectors transduce nondividing hematopoietic cells more efficiently than other currently available vector systems. Here we report the results of human hematopoietic cell gene transfer using lentiviral vectors based upon human immunodeficiency virus (HIV-1) and equine infectious anemia virus (EIAV). EIAV is a nonprimate lentivirus and is severely restricted in its host range to horses and closely related equines. We employed the EIAV vector system to test for gene transfer to human Fanconi anemia (FA) hematopoietic cells by comparison with HIV-1- and Moloney murine leukemia virus-based systems. Fanconi anemia is characterized by bone marrow failure secondary to stem cell dysfunction. Fanconi anemia group C EBV-transformed lymphoblasts were transduced with all three viral vectors. Phenotypic correction of FA cells, as measured by mitomycin C drug resistance, was observed with a similar efficiency in all vector systems. This is the first description of lentiviral correction of FA cells and suggests that lentiviral vectors may be useful for FA gene transfer.
引用
收藏
页码:485 / 490
页数:6
相关论文
共 40 条
[1]   High-efficiency gene transfer into CD34(+) cells with a human immunodeficiency virus type 1-based retroviral vector pseudotyped with vesicular stomatitis virus envelope glycoprotein G [J].
Akkina, RK ;
Walton, RM ;
Chen, ML ;
Li, QX ;
Planelles, V ;
Chen, ISY .
JOURNAL OF VIROLOGY, 1996, 70 (04) :2581-2585
[2]   Marking and gene expression by a lentivirus vector in transplanted human and nonhuman primate CD34+ cells [J].
An, DS ;
Wersto, RP ;
Agricola, BA ;
Metzger, ME ;
Lu, S ;
Amado, RG ;
Chen, ISY ;
Donahue, RE .
JOURNAL OF VIROLOGY, 2000, 74 (03) :1286-1295
[3]  
Blomer U, 1997, J VIROL, V71, P6641
[4]   VESICULAR STOMATITIS-VIRUS G GLYCOPROTEIN PSEUDOTYPED RETROVIRAL VECTORS - CONCENTRATION TO VERY HIGH-TITER AND EFFICIENT GENE-TRANSFER INTO MAMMALIAN AND NONMAMMALIAN CELLS [J].
BURNS, JC ;
FRIEDMANN, T ;
DRIEVER, W ;
BURRASCANO, M ;
YEE, JK .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1993, 90 (17) :8033-8037
[5]   Stable transduction of quiescent CD34+CD38- human hematopoietic cells by HIV-1-based lentiviral vectors [J].
Case, SS ;
Price, MA ;
Jordan, CT ;
Yu, XJ ;
Wang, LJ ;
Bauer, G ;
Haas, DL ;
Xu, DK ;
Stripecke, R ;
Naldini, L ;
Kohn, DB ;
Crooks, GM .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1999, 96 (06) :2988-2993
[6]   Lentiviral vector transduction of hematopoietic stem cells that mediate long-term reconstitution of lethally irradiated mice [J].
Chen, WY ;
Wu, XY ;
Levasseur, DN ;
Liu, HM ;
Lai, LL ;
Kappes, JC ;
Townes, TM .
STEM CELLS, 2000, 18 (05) :352-359
[7]   SINGLE-STEP METHOD OF RNA ISOLATION BY ACID GUANIDINIUM THIOCYANATE PHENOL CHLOROFORM EXTRACTION [J].
CHOMCZYNSKI, P ;
SACCHI, N .
ANALYTICAL BIOCHEMISTRY, 1987, 162 (01) :156-159
[8]   MOLECULAR-BASIS OF THE PATHOBIOLOGY OF LENTIVIRUSES [J].
CLEMENTS, JE ;
PAYNE, SL .
VIRUS RESEARCH, 1994, 32 (02) :97-109
[9]   THE CELL-CYCLE OF LYMPHOCYTES IN FANCONI ANEMIA [J].
DUTRILLAUX, B ;
AURIAS, A ;
DUTRILLAUX, AM ;
BURIOT, D ;
PRIEUR, M .
HUMAN GENETICS, 1982, 62 (04) :327-332
[10]   Human cord blood CD34+CD38- cell transduction via lentivirus-based gene transfer vectors [J].
Evans, JT ;
Kelly, PF ;
O'Neill, E ;
Garcia, JV .
HUMAN GENE THERAPY, 1999, 10 (09) :1479-1489