ENGRAFTMENT OF GENE-MODIFIED UMBILICAL-CORD BLOOD-CELLS IN NEONATES WITH ADENOSINE-DEAMINASE DEFICIENCY

被引：475

作者：

KOHN, DB

WEINBERG, KI

NOLTA, JA

HEISS, LN

LENARSKY, C

CROOKS, GM

HANLEY, ME

ANNETT, G

BROOKS, JS

ELKHOUREIY, A

LAWRENCE, K

WELLS, S

MOEN, RC

BASTIAN, J

WILLIAMSHERMAN, DE

ELDER, M

WARA, D

BOWEN, T

HERSHFIELD, MS

MULLEN, CA

BLAESE, RM

PARKMAN, R

机构：

[1] GENET THERAPY INC,GAITHERSBURG,MD 20878

[2] CHILDRENS HOSP & HLTH CTR,SAN DIEGO,CA 92123

[3] UNIV CALIF SAN FRANCISCO,DEPT PEDIAT,SAN FRANCISCO,CA 94143

[4] DUKE UNIV,MED CTR,DEPT MED,DURHAM,NC 27710

[5] NIH,NATL CTR HUMAN GENOME RES,BETHESDA,MD 20892

来源：

NATURE MEDICINE | 1995年 / 1卷 / 10期

关键词：

D O I：

10.1038/nm1095-1017

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Haematopoietic stem cells in umbilical cord blood are an attractive target for gene therapy of inborn errors of metabolism. Three neonates with severe combined immunodeficiency were treated by retroviral-mediated transduction of the CD34+ cells from their umbilical cord blood with a normal human adenosine deaminase complementary DNA followed by autologous transplantation. The continued presence and expression of the introduced gene in leukocytes from bone marrow and peripheral blood for 18 months demonstrates that umbilical cord blood cells may be genetically modified with retroviral vectors and engrafted in neonates for gene therapy.

引用

页码：1017 / 1023

页数：7

共 31 条

[1] PARADOXICAL EXPRESSION OF ADENOSINE-DEAMINASE IN T-CELLS CULTURED FROM A PATIENT WITH ADENOSINE-DEAMINASE DEFICIENCY AND COMBINED IMMUNODEFICIENCY [J].