Gene therapy for primary immunodeficiencies: part 1

被引：59

作者：

Cavazzana-Calvo, Marina ^{[1
,2
,4
,5
]}

Fischer, Alain ^{[1
,3
,4
]}

Hacein-Bey-Abina, Salima ^{[2
,4
,5
]}

Aiuti, Alessandro ^{[6
,7
]}

机构：

[1] INSERM, U768, Paris, France

[2] Hop Univ Necker Enfants Malad, AP HP, Dept Biotherapie, Paris, France

[3] Hop Univ Necker Enfants Malad, Unite Immunohematol Pediat, Paris, France

[4] Univ Paris 05, Sorbonne Paris Cite, IMAGINE Inst, Paris, France

[5] INSERM APHP, CIC Biotherapie GHU Ouest, Paris, France

[6] San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy

[7] Univ Roma Tor Vergata, Childrens Hosp Bambino Gesu, Dept Pediat, Rome, Italy

来源：

CURRENT OPINION IN IMMUNOLOGY | 2012年 / 24卷 / 05期

基金：

欧洲研究理事会;

关键词：

ADENOSINE-DEAMINASE DEFICIENCY; IN-VIVO; SCID-X1; CELLS; RECONSTITUTION; ARTEMIS;

D O I：

10.1016/j.coi.2012.08.008

中图分类号：

R392 [医学免疫学]; Q939.91 [免疫学];

学科分类号：

100102 ;

摘要：

Over 60 patients affected by SCID due to IL2RG deficiency (SCID-X1) or adenosine deaminase (ADA)-SCID have received hematopoietic stem cell gene therapy in the past 15 years using gammaretroviral vectors, resulting in immune reconstitution and clinical benefit in the majority of them. However, the occurrence of insertional oncogenesis in the SCID-X1 trials has led to the development of new clinical trials based on integrating vectors with improved safety design as well as investigation on new technologies for highly efficient gene targeting and site-specific gene editing. Here we will present the experience and perspectives of gene therapy for SCID-X1 and ADA-SCID and discuss the pros and cons of gene therapy in comparison to allogeneic transplantation.

引用

页码：580 / 584

页数：5

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