X连锁重症联合免疫缺陷的基因治疗：历史成果与现状 - Suppr

Gene therapy for X-linked severe combined immunodeficiency: Historical outcomes and current status.

作者信息

Pai Sung-Yun, Thrasher Adrian J

机构信息

Division of Hematology-Oncology, Boston Children's Hospital and Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Mass.

University College London Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.

出版信息

J Allergy Clin Immunol. 2020 Aug;146(2):258-261. doi: 10.1016/j.jaci.2020.05.055. Epub 2020 Jun 16.

DOI:10.1016/j.jaci.2020.05.055

PMID:32561390

Abstract

摘要

相似文献

Gene therapy for X-linked severe combined immunodeficiency: Historical outcomes and current status.X连锁重症联合免疫缺陷的基因治疗：历史成果与现状

J Allergy Clin Immunol. 2020 Aug;146(2):258-261. doi: 10.1016/j.jaci.2020.05.055. Epub 2020 Jun 16.

Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency.基因疗法可改善患有X连锁重症联合免疫缺陷的青春期前儿童的免疫功能。

Blood. 2007 Jul 1;110(1):67-73. doi: 10.1182/blood-2006-11-058933. Epub 2007 Mar 16.

Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?X连锁重症联合免疫缺陷的基因治疗：我们目前的进展如何？

Hum Gene Ther. 2016 Feb;27(2):108-16. doi: 10.1089/hum.2015.137.

Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.用于X连锁重症联合免疫缺陷基因治疗的自失活γ逆转录病毒载体

Mol Ther. 2008 Mar;16(3):590-8. doi: 10.1038/sj.mt.6300393. Epub 2008 Jan 8.

New Gene Therapy Potential Cure for "Bubble Boy Disease": An experimental gene therapy has allowed children with SCID-1X to develop fully functioning immune systems.新型基因疗法有望治愈“泡泡男孩病”：一种实验性基因疗法使患有X连锁重症联合免疫缺陷病（SCID-1X）的儿童能够发育出功能完全正常的免疫系统。

Am J Med Genet A. 2019 Jul;179(7):1114-1115. doi: 10.1002/ajmg.a.40453.

Restoring T and B cell generation in X-linked severe combined immunodeficiency mice through hematopoietic stem cells adenine base editing.通过造血干细胞腺嘌呤碱基编辑恢复 X 连锁严重联合免疫缺陷小鼠中的 T 和 B 细胞生成。

Mol Ther. 2024 Jun 5;32(6):1658-1671. doi: 10.1016/j.ymthe.2024.03.028. Epub 2024 Mar 26.

New insights and unresolved issues regarding insertional mutagenesis in X-linked SCID gene therapy.关于X连锁严重联合免疫缺陷病基因治疗中插入诱变的新见解与未解决问题。

Mol Ther. 2007 Nov;15(11):1910-6. doi: 10.1038/sj.mt.6300297. Epub 2007 Aug 28.

SASH3 variants cause a novel form of X-linked combined immunodeficiency with immune dysregulation.SASH3 变异导致一种新型的 X 连锁联合免疫缺陷伴免疫失调。

Blood. 2021 Sep 23;138(12):1019-1033. doi: 10.1182/blood.2020008629.

A novel common gamma chain mutation in a Chinese family with X-linked severe combined immunodeficiency (X-SCID; T(-)NK(-)B(+)).一个患有X连锁重症联合免疫缺陷（X-SCID；T(-)NK(-)B(+)）的中国家庭中的一种新型常见γ链突变。

Immunogenetics. 2015 Nov;67(11-12):629-39. doi: 10.1007/s00251-015-0871-0. Epub 2015 Sep 26.

Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease.X 连锁严重联合免疫缺陷病患者基因治疗后的免疫重建。

Front Immunol. 2020 Nov 27;11:608653. doi: 10.3389/fimmu.2020.608653. eCollection 2020.

引用本文的文献

CRISPR/Cas-Based Ex Vivo Gene Therapy and Lysosomal Storage Disorders: A Perspective Beyond Cas9.基于CRISPR/Cas的体外基因治疗与溶酶体贮积症：超越Cas9的视角

Cells. 2025 Jul 25;14(15):1147. doi: 10.3390/cells14151147.

The clinical and socioeconomic aspects of t-cell receptor excision circle based newborn screening for severe combined immunodeficiency in Southeast and East Asia.基于T细胞受体切除环的新生儿筛查在东南亚和东亚地区诊断重症联合免疫缺陷的临床及社会经济方面

Front Immunol. 2025 May 19;16:1549768. doi: 10.3389/fimmu.2025.1549768. eCollection 2025.

Preclinical IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency disease.使用自体造血干细胞的临床前白细胞介素2受体γ链基因疗法作为X连锁重症联合免疫缺陷病的一种有效且安全的治疗方法。

Genes Dis. 2024 Nov 6;12(3):101445. doi: 10.1016/j.gendis.2024.101445. eCollection 2025 May.

[Advances in gene therapy for inborn errors of immunity].[先天性免疫缺陷病的基因治疗进展]

Zhongguo Dang Dai Er Ke Za Zhi. 2024 Aug 15;26(8):865-870. doi: 10.7499/j.issn.1008-8830.2404027.

A novel X-linked mutation in IL2RG associated with early-onset inflammatory bowel disease: a case report of twin brothers.一个新的与早发性炎症性肠病相关的 X 连锁 IL2RG 突变：一对双胞胎兄弟的病例报告。

J Med Case Rep. 2023 Jul 18;17(1):307. doi: 10.1186/s13256-023-04049-y.

Correcting inborn errors of immunity: From viral mediated gene addition to gene editing.纠正先天性免疫缺陷：从病毒介导的基因添加到基因编辑。

Semin Immunol. 2023 Mar;66:101731. doi: 10.1016/j.smim.2023.101731. Epub 2023 Feb 28.

Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime Editing.CRISPR/Cas9 和 Prime Editing 在 X 连锁联合免疫缺陷症体性回复嵌合体基因治疗中的挑战。

Genes (Basel). 2022 Dec 13;13(12):2348. doi: 10.3390/genes13122348.

Gene therapy for inborn errors of immunity: past, present and future.免疫缺陷性遗传病的基因治疗：过去、现在和未来。

Nat Rev Immunol. 2023 Jun;23(6):397-408. doi: 10.1038/s41577-022-00800-6. Epub 2022 Nov 25.

Development and clinical translation of gene therapy.基因治疗的研发与临床转化

Comput Struct Biotechnol J. 2022 Jun 11;20:2986-3003. doi: 10.1016/j.csbj.2022.06.015. eCollection 2022.

X-linked SCID with a rare mutation.伴有罕见突变的X连锁重症联合免疫缺陷病

Allergy Asthma Clin Immunol. 2021 Oct 11;17(1):107. doi: 10.1186/s13223-021-00605-7.

文献检索

告别复杂PubMed语法，用中文像聊天一样搜索，搜遍4000万医学文献。AI智能推荐，让科研检索更轻松。

立即免费搜索

文件翻译

保留排版，准确专业，支持PDF/Word/PPT等文件格式，支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述，25分钟生成高质量综述，智能提取关键信息，辅助科研写作。

立即免费体验

Gene therapy for X-linked severe combined immunodeficiency: Historical outcomes and current status.

作者信息

机构信息

出版信息

相似文献

引用本文的文献

文献检索

文件翻译

深度研究

Suppr 超能文献

相似文献

引用本文的文献