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子宫内基因治疗原发性免疫缺陷病。

In Utero Gene Therapy for Primary Immunodeficiencies.

机构信息

Department of Obstetrics, Gynecology, and Reproductive Services, University of California, San Francisco, California.

出版信息

Clin Obstet Gynecol. 2021 Dec 1;64(4):886-897. doi: 10.1097/GRF.0000000000000652.

Abstract

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.

摘要

原发性免疫缺陷病 (PIDs) 已成为基因治疗的主要目标,因为其发病率、死亡率和单基因病因。鉴于基因治疗越早实施效果越好,因此胎儿基因治疗可能是治疗 PIDs 的一个重要未来方向。在这一章中,将回顾几种 PID 的现有治疗方法,以及 PID 基因治疗的历史和现状。然后将讨论宫内基因治疗的可能性。

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