Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID

被引：23

作者：

Aiuti, Alessandro ^{[1
,2
,3
]}

Brigida, Immacolata ^{[1
,2
]}

Ferrua, Francesca ^{[1
,2
]}

Cappelli, Barbara ^{[1
,2
]}

Chiesa, Robert ^{[1
,2
]}

Marktel, Sarah ^{[1
,2
]}

Roncarolo, Maria-Grazia ^{[1
,2
,4
]}

机构：

[1] Sci Inst HS Raffaele, San Raffaele Telethon Inst Gene Therapy HSR TIGET, I-20132 Milan, Italy

[2] Sci Inst HS Raffaele, Pediat Immunohematol & Bone Marrow Transplant Uni, I-20132 Milan, Italy

[3] Univ Roma Tor Vergata, Rome, Italy

[4] Univ Vita Salute, Milan, Italy

来源：

IMMUNOLOGIC RESEARCH | 2009年 / 44卷 / 1-3期

关键词：

Gene therapy; Hematopoietic stem cell; Bone marrow; Severe combined immunodeficiency; Retroviral vector; SEVERE COMBINED IMMUNODEFICIENCY; BONE-MARROW-TRANSPLANTATION; INSERTIONAL MUTAGENESIS; CD34(+) CELLS; CORD BLOOD; IMMUNE RECONSTITUTION; LENTIVIRAL VECTORS; MOUSE MODEL; ADA; LYMPHOCYTES;

D O I：

10.1007/s12026-009-8107-8

中图分类号：

R392 [医学免疫学]; Q939.91 [免疫学];

学科分类号：

100102 ;

摘要：

Gene therapy is a highly attractive strategy for many types of inherited disorders of the immune system. Adenosine deaminase (ADA) deficient-severe combined immunodeficiency (SCID) has been the target of several clinical trials based on the use of hematopoietic stem/progenitor cells engineered with retroviral vectors. The introduction of a low intensity conditioning regimen has been a crucial factor in achieving stable engrafment of hematopoietic stem cells and therapeutic levels of ADA-expressing cells. Recent studies have demonstrated that gene therapy for ADA-SCID has favorable safety profile and is effective in restoring normal purine metabolism and immune functions. Stem cell gene therapy combined with appropriate conditioning regimens might be extended to other genetic disorders of the hematopoietic system.

引用

页码：150 / 159

页数：10

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