Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: Improved retroviral vectors for clinical trials

被引：25

作者：

Onodera, M

Nelson, DM

Sakiyama, Y

Candotti, F

Blaese, RM

机构：

[1] Hokkaido Univ, Sch Med, Dept Pediat, Sapporo, Hokkaido 060, Japan

[2] Natl Human Genome Res Inst, Clin Gene Therapy Branch, NIH, Bethesda, MD USA

[3] CREST, Japan Sci & Technol Corp, Tsukuba, Ibaraki, Japan

来源：

ACTA HAEMATOLOGICA | 1999年 / 101卷 / 02期

关键词：

gene therapy; retroviral vector; severe combined immunodeficiency; adenosine deaminase; stem cells; hematopoietic; T lymphocytes; peripheral;

D O I：

10.1159/000040930

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Severe combined immunodeficiency (SCID) caused by adenosine deaminase deficiency (ADA-) is the first genetic disorder to be treated with gene therapy. Since 1990 when the first trial started for 2 patients with ADA-SCID, five clinical trials enrolling 11 patients have been conducted with different clinical approaches and the results obtained from these trials have recently been reported. According to these reports, T cell-directed gene transfer was useful in the treatment of ADA- SCID whereas the retroviral-mediated gene transfer to hematopoietic stem cells was insufficient for achievement of clinical benefits. This chapter reviews several crucial problems inherent in the current retroviral technology based on the clinical data observed in these pioneering ADA gene therapy trials and presents our new retroviral vector system for the next stem cell gene therapy.

引用

页码：89 / 96

页数：8

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