Gene therapy for genetic haematological disorders and immunodeficiencies

被引：35

作者：

Kohn, DB

机构：

[1] Childrens Hosp Los Angeles, Div Res Immunol Bone Marrow Transplantat, Los Angeles, CA 90027 USA

[2] Univ So Calif, Keck Sch Med, Los Angeles, CA 90033 USA

来源：

JOURNAL OF INTERNAL MEDICINE | 2001年 / 249卷 / 04期

关键词：

gene therapy; haematopoietic stem cells; haemoglobinopathies; immune deficiency syndromes; lysosomal storage diseases; retroviral vectors;

D O I：

10.1046/j.1365-2796.2001.00803.x

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Gene transfer and autologous transplantation of haematopoietic stem cells (HSCs) from patients with genetic haematological disorders and immunodeficiencies could provide the same benefits as allogeneic HSC transplantation, without the attendant immunological complications. Inefficient gene delivery to human HSCs has imposed the major limitation to successful application of gene therapy. A recently reported clinical trial of gene transfer into HSCs of infants with X-linked severe combined immunodeficiency (SCID) has achieved immune restoration because of the selective outgrowth of the gene-corrected lymphocytes, Newer methods for manipulating HSCs may lead to efficacy for other disorders. The problems and progress in this area are reviewed herein.

引用

页码：379 / 390

页数：12

共 69 条

[1] Scaffold attachment region-mediated enhancement of retroviral vector expression in primary T cells [J].