Liver-directed gene transfer vectors

被引:72
作者
Ferry, N
Heard, JM
机构
[1] Inst Pasteur, Lab Retrovirus & Transfert Genet, F-75015 Paris, France
[2] Lab Therapie Gen, F-44035 Nantes, France
关键词
D O I
10.1089/hum.1998.9.14-1975
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
The ultimate goal of liver-directed gene therapy for genetic diseases is the stable expression of a therapeutic transgene in a significant proportion of hepatocytes. This article considers the various liver-directed gene transfer procedures studied so far. Performances and limitations of currently available vector systems are discussed with respect to their clinical relevance. Although some improvements have been reported, naked DNA and nonviral gene transfer vectors induce transient expression in only a limited number of cells. Clinical applications of retrovirus-mediated gene transfer are hampered by the need to induce hepatocyte division. First-generation adenovirus vectors are highly efficient; however, they induce an immune response leading to the rapid rejection of transduced cells. Promising new vector systems have emerged, including gutless adenovirus vectors, adenoassociated vectors, and lentivirus vectors. However, these systems are still poorly documented and their relevance to liver-directed gene therapy must be confirmed.
引用
收藏
页码:1975 / 1981
页数:7
相关论文
共 96 条
[51]   IN-VIVO HEPATIC GENE-THERAPY - COMPLETE ALBEIT TRANSIENT CORRECTION OF FACTOR-IX DEFICIENCY IN HEMOPHILIA-B DOGS [J].
KAY, MA ;
LANDEN, CN ;
ROTHENBERG, SR ;
TAYLOR, LA ;
LELAND, F ;
WIEHLE, S ;
FANG, BL ;
BELLINGER, D ;
FINEGOLD, M ;
THOMPSON, AR ;
READ, M ;
BRINKHOUS, KM ;
WOO, SLC .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1994, 91 (06) :2353-2357
[52]   Transient immunomodulation with anti-CD40 ligand antibody and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver [J].
Kay, MA ;
Meuse, L ;
Gown, AM ;
Linsley, P ;
Hollenbaugh, D ;
Aruffo, A ;
Ochs, HD ;
Wilson, CB .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1997, 94 (09) :4686-4691
[53]   IN-VIVO GENE-THERAPY OF HEMOPHILIA-B - SUSTAINED PARTIAL CORRECTION IN FACTOR-IX-DEFICIENT DOGS [J].
KAY, MA ;
ROTHENBERG, S ;
LANDEN, CN ;
BELLINGER, DA ;
LELAND, F ;
TOMAN, C ;
FINEGOLD, M ;
THOMPSON, AR ;
READ, MS ;
BRINKHOUS, KM ;
WOO, SLC .
SCIENCE, 1993, 262 (5130) :117-119
[54]   HEPATIC GENE-THERAPY - PERSISTENT EXPRESSION OF HUMAN ALPHA-1-ANTITRYPSIN IN MICE AFTER DIRECT GENE DELIVERY INVIVO [J].
KAY, MA ;
LI, QT ;
LIU, TJ ;
LELAND, F ;
TOMAN, C ;
FINEGOLD, M ;
WOO, SLC .
HUMAN GENE THERAPY, 1992, 3 (06) :641-647
[55]   Highly efficient retrovirus-mediated gene transfer into rat hepatocytes in vivo [J].
Kitten, O ;
Cosset, FL ;
Ferry, N .
HUMAN GENE THERAPY, 1997, 8 (12) :1491-1494
[56]   Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors [J].
Koeberl, DD ;
Alexander, IE ;
Halbert, CL ;
Russell, DW ;
Miller, AD .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1997, 94 (04) :1426-1431
[57]  
KOZARSKY KF, 1994, J BIOL CHEM, V269, P13695
[58]   In vivo correction with recombinant adenovirus of 4-hydroxyphenylpyruvic acid dioxygenase deficiencies in strain III mice [J].
Kubo, S ;
Kiwaki, K ;
Awata, H ;
Katoh, H ;
Kanegae, Y ;
Saito, I ;
Yamamoto, T ;
Miyazaki, J ;
Matsuda, I ;
Endo, F .
HUMAN GENE THERAPY, 1997, 8 (01) :65-71
[59]   Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy [J].
Le, MT ;
Okuyama, T ;
Cai, SR ;
Kennedy, SC ;
Bowling, WM ;
Flye, MW ;
Ponder, KP .
BLOOD, 1997, 89 (04) :1254-1259
[60]   RETROVIRAL GENE-TRANSFER INTO PRIMARY HEPATOCYTES - IMPLICATIONS FOR GENETIC THERAPY OF LIVER-SPECIFIC FUNCTIONS [J].
LEDLEY, FD ;
DARLINGTON, GJ ;
HAHN, T ;
WOO, SLC .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1987, 84 (15) :5335-5339