GENE-THERAPY FOR METABOLIC DISORDERS

被引：93

作者：

KAY, MA

WOO, SLC

机构：

[1] HOWARD HUGHES MED INST,HOUSTON,TX 77030

[2] BAYLOR COLL MED,HOUSTON,TX 77030

来源：

TRENDS IN GENETICS | 1994年 / 10卷 / 07期

关键词：

D O I：

10.1016/0168-9525(94)90173-2

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Most diseases caused by genetic deficiencies could, in theory, be treated by the introduction and expression of a normal gene into an appropriate target tissue. It seems likely that gene therapy strategies for most metabolic disorders will not require strict gene regulation, as a fraction of the normal levels of gene activity could result in amelioration or significant improvement in the clinical outcome. Gene therapy is making rapid progress towards the goal of treating various disorders: here, we summarize the state of gene therapy for metabolic disorders.

引用

页码：253 / 257

页数：5

共 49 条

[31] MIYANOHARA A, 1992, NEW BIOL, V4, P238
[32] CORRECTION OF LYSOSOMAL STORAGE IN THE LIVER AND SPLEEN OF MPS-VII MICE BY IMPLANTATION OF GENETICALLY-MODIFIED SKIN FIBROBLASTS
MOULLIER, P
BOHL, D
HEARD, JM
DANOS, O
[J]. NATURE GENETICS, 1993, 4 (02) : 154 - 159
[33] MUYCZKA N, 1992, CURR TOP MICROBIOL I, V158, P97
[34] NOLA JA, 1992, J CLIN INVEST, V90, P342
[35] OSHASHI T, 1992, P NATL ACAD SCI USA, V89, P11332
[36] LONG-TERM INVIVO EXPRESSION OF RETROVIRUS-MEDIATED GENE-TRANSFER IN MOUSE FIBROBLAST IMPLANTS
SCHARFMANN, R
AXELROD, JH
VERMA, IM
[J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1991, 88 (11) : 4626 - 4630
[37] SCHUENING FG, 1992, CURR TOP MICROBIOL, V177, P237
[38] GAUCHER DISEASE IN THE NEONATE - A DISTINCT GAUCHER PHENOTYPE IS ANALOGOUS TO A MOUSE MODEL CREATED BY TARGETED DISRUPTION OF THE GLUCOCEREBROSIDASE GENE
SIDRANSKY, E
SHERER, DM
GINNS, EL
[J]. PEDIATRIC RESEARCH, 1992, 32 (04) : 494 - 498
[39] ADENOVIRUS-MEDIATED EXPRESSION OF THERAPEUTIC PLASMA-LEVELS OF HUMAN FACTOR-IX IN MICE
SMITH, TAG
MEHAFFEY, MG
KAYDA, DB
SAUNDERS, JM
YEI, S
TRAPNELL, BC
MCCLELLAND, A
KALEKO, M
[J]. NATURE GENETICS, 1993, 5 (04) : 397 - 402
[40] EVALUATION OF THE TRANSFER AND EXPRESSION IN MICE OF AN ENZYME-ENCODING GENE USING A HUMAN ADENOVIRUS VECTOR
STRATFORDPERRICAUDET, LD
LEVRERO, M
CHASSE, JF
PERRICAUDET, M
BRIAND, P
[J]. HUMAN GENE THERAPY, 1990, 1 (03) : 241 - 256

← 1 2 3 4 5 →