Bone Marrow Transplantation and Alternatives for Adenosine Deaminase Deficiency

被引：37

作者：

Gaspar, H. Bobby ^{[1
]}

机构：

[1] UCL Inst Child Hlth, Ctr Immunodeficiency, Mol Immunol Unit, London WC1N 1EH, England

来源：

IMMUNOLOGY AND ALLERGY CLINICS OF NORTH AMERICA | 2010年 / 30卷 / 02期

关键词：

Adenosine deaminase; Severe combined immunodeficiency; Stem cell gene therapy; Enzyme replacement therapy; SEVERE COMBINED IMMUNODEFICIENCY; ENZYME REPLACEMENT THERAPY; CELL GENE-THERAPY; ADA-SCID PATIENTS; POLYETHYLENE-GLYCOL; BEHAVIORAL ABNORMALITIES; IMMUNE-DEFICIENCY; CIRCULATING LIFE; MICE; RECONSTITUTION;

D O I：

10.1016/j.iac.2010.01.002

中图分类号：

R392 [医学免疫学];

学科分类号：

100102 ;

摘要：

Adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID) comprises approximately 10% to 15% of all cases of SCID. The clinical effects of ADA deficiency are manifest most dramatically in the immune system, where it leads to severe lymphopenia. Although hematopoietic stem cell transplantation remains the mainstay of treatment for ADA-deficient SCID, 2 other treatment options are available, namely enzyme replacement therapy with PEG-ADA and autologous hematopoietic stem cell gene therapy. In this article the author reviews the available data on treatment by these different options, and offers an overview on when each of the different treatment options should be used.

引用

页码：221 / +

页数：18

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