T-LYMPHOCYTE-DIRECTED GENE-THERAPY FOR ADA(-) SCID - INITIAL TRIAL RESULTS AFTER 4 YEARS

被引：1017

作者：

BLAESE, RM

CULVER, KW

MILLER, AD

CARTER, CS

FLEISHER, T

CLERICI, M

SHEARER, G

CHANG, L

CHIANG, YW

TOLSTOSHEV, P

GREENBLATT, JJ

ROSENBERG, SA

KLEIN, H

BERGER, M

MULLEN, CA

RAMSEY, WJ

MUUL, L

MORGAN, RA

ANDERSON, WF

机构：

[1] NCI, BETHESDA, MD 20892 USA

[2] FRED HUTCHINSON CANC RES CTR, SEATTLE, WA 98104 USA

[3] NIH, CTR CLIN, BETHESDA, MD 20892 USA

[4] NHLBI, BETHESDA, MD 20892 USA

[5] GENET THERAPY, GAITHERSBURG, MD 20878 USA

[6] CASE WESTERN RESERVE UNIV, SCH MED, DEPT PEDIAT, CLEVELAND, OH 44106 USA

来源：

SCIENCE | 1995年 / 270卷 / 5235期

关键词：

D O I：

10.1126/science.270.5235.475

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：

In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA(-) SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.

引用

页码：475 / 480

页数：6

共 75 条

[1] NORMAL AND MUTANT HUMAN ADENOSINE-DEAMINASE GENES [J].