In Utero Gene Therapy for Primary Immunodeficiencies

被引：3

作者：

Mardy, Anne H. ^{[1
]}

Norton, Mary E. ^{[1
]}

机构：

[1] Univ Calif San Francisco, Dept Obstet Gynecol & Reprod Serv, San Francisco, CA 94143 USA

来源：

CLINICAL OBSTETRICS AND GYNECOLOGY | 2021年 / 64卷 / 04期

关键词：

gene therapy; in utero gene therapy; SCID; primary immunodeficiencies; Wiskott-Aldrich syndrome; chronic granulomatous disease; STEM-CELL TRANSPLANTATION; CHRONIC GRANULOMATOUS-DISEASE; BONE-MARROW-TRANSPLANTATION; ADENOSINE-DEAMINASE DEFICIENCY; WISKOTT-ALDRICH SYNDROME; SENSORINEURAL DEAFNESS; INTERNATIONAL UNION; INBORN-ERRORS; BLOOD-CELLS; FETAL;

D O I：

10.1097/GRF.0000000000000652

中图分类号：

R71 [妇产科学];

学科分类号：

100211 ;

摘要：

Primary immunodeficiencies (PIDs) have become a prime target for gene therapy given the morbidity, mortality, and the single gene etiology. Given that outcomes are better the earlier gene therapy is implemented, it is possible that fetal gene therapy may be an important future direction for the treatment of PIDs. In this chapter, the current treatments available for several PIDs will be reviewed, as well as the history and current status of gene therapy for PIDs. The possibility of in utero gene therapy as a possibility will then be discussed.

引用

页码：886 / 897

页数：12

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