Sustainable cutaneous gene delivery

被引:70
作者
Deng, H
Lin, Q
Khavari, PA
机构
[1] VA PALO ALTO HLTH CARE SYST,PALO ALTO,CA 94304
[2] STANFORD UNIV,SCH MED,DEPT DERMATOL,STANFORD,CA 94305
关键词
gene therapy; retroviral vectors;
D O I
10.1038/nbt1297-1388
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
Durable gene delivery to human skin is necessary for lasting correction of human genetic skin disease. Current cutaneous gene-delivery strategies, however, have achieved only transient gene expression, often only within a small percentage of tissue cells. The recent inability to sustain phenotypic correction of human genetic skin disease due to loss of therapeutic gene expression in regenerated epidermal tissue has highlighted this current limitation. In an effort to surmount this problem, we have generated gene delivery vectors that produce more durable gene delivery in human skin tissue in vivo.
引用
收藏
页码:1388 / 1391
页数:4
相关论文
共 33 条
[1]  
[Anonymous], 1996, B MED ETHICS
[2]  
CHAKRABORTY AK, 1993, FASEB J, V7, P91
[3]   LACK OF EXPRESSION FROM A RETROVIRAL VECTOR AFTER TRANSDUCTION OF MURINE HEMATOPOIETIC STEM-CELLS IS ASSOCIATED WITH METHYLATION IN-VIVO [J].
CHALLITA, PM ;
KOHN, DB .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1994, 91 (07) :2567-2571
[4]   THE GENETIC-BASIS OF EPIDERMOLYTIC HYPERKERATOSIS - A DISORDER OF DIFFERENTIATION-SPECIFIC EPIDERMAL KERATIN GENES [J].
CHENG, J ;
SYDER, AJ ;
YU, QC ;
LETAI, A ;
PALLER, AS ;
FUCHS, E .
CELL, 1992, 70 (05) :811-819
[5]   Sustainability of keratinocyte gene transfer and cell survival in vivo [J].
Choate, KA ;
Khavari, PA .
HUMAN GENE THERAPY, 1997, 8 (08) :895-901
[6]   Transglutaminase 1 delivery to lamellar ichthyosis keratinocytes [J].
Choate, KA ;
Kinsella, TM ;
Williams, ML ;
Nolan, GP ;
Khavari, PA .
HUMAN GENE THERAPY, 1996, 7 (18) :2247-2253
[7]   Corrective gene transfer in the human skin disorder lamellar ichthyosis [J].
Choate, KA ;
Medalie, DA ;
Morgan, JR ;
Khavari, PA .
NATURE MEDICINE, 1996, 2 (11) :1263-1267
[8]   A FUNCTION FOR KERATINS AND A COMMON THREAD AMONG DIFFERENT TYPES OF EPIDERMOLYSIS-BULLOSA SIMPLEX DISEASES [J].
COULOMBE, PA ;
HUTTON, ME ;
VASSAR, R ;
FUCHS, E .
JOURNAL OF CELL BIOLOGY, 1991, 115 (06) :1661-1674
[9]   EXPRESSION PATTERN OF THE BULLOUS PEMPHIGOID-180 ANTIGEN IN NORMAL AND NEOPLASTIC EPITHELIA [J].
FAIRLEY, JA ;
HEINTZ, PW ;
NEUBURG, M ;
DIAZ, LA ;
GIUDICE, GJ .
BRITISH JOURNAL OF DERMATOLOGY, 1995, 133 (03) :385-391
[10]   A NEW FAMILY OF MURINE RETROVIRAL VECTORS WITH EXTENDED MULTIPLE CLONING SITES FOR GENE INSERTION [J].
FAUSTINELLA, F ;
KWON, HC ;
SERRANO, F ;
BELMONT, JW ;
CASKEY, CT ;
AGUILARCORDOVA, E .
HUMAN GENE THERAPY, 1994, 5 (03) :307-312