[先天性免疫错误基因疗法的进展]。

Q3 Medicine 中国当代儿科杂志 Pub Date : 2024-08-15 DOI:10.7499/j.issn.1008-8830.2404027

Ting Li, Hong-Mei Song

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引用次数: 0

摘要

先天性免疫错误（IEI）是由免疫系统结构或功能缺陷引起的一组多种多样的疾病，涉及先天性免疫和适应性免疫。2022 年更新的 IEI 分类包括 485 种不同的疾病，分为十大疾病群。随着分子生物学的快速发展，许多 IEI 的特异性发病机制已被揭示，这使得基因治疗在这类疾病的临床前和临床研究中成为可能。本文回顾了基因疗法治疗 IEI 的进展，旨在提高人们对这类疾病的认识和了解。

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[Advances in gene therapy for inborn errors of immunity].

Inborn errors of immunity (IEI) are a diverse group of disorders caused by defects in immune system structure or function, involving both innate and adaptive immunity. The 2022 update of the IEI classification includes 485 distinct disorders, categorized into ten major disease groups. With the rapid development of molecular biology, the specific pathogenesis of many IEI has been revealed, making gene therapy possible in preclinical and clinical research of this type of disease. This article reviews the advancements in gene therapy for IEI, aiming to increase awareness and understanding of these disorders.

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来源期刊

中国当代儿科杂志 Medicine-Pediatrics, Perinatology and Child Health

CiteScore

1.50

自引率

0.00%

发文量

5006

期刊介绍： The Chinese Journal of Contemporary Pediatrics (CJCP) is a peer-reviewed open access periodical in the field of pediatrics that is sponsored by the Central South University/Xiangya Hospital of Central South University and under the auspices of the Ministry of Education of China. It is cited as a source in the scientific and technological papers of Chinese journals, the Chinese Science Citation Database (CSCD), and is one of the core Chinese periodicals in the Peking University Library. CJCP has been indexed by MEDLINE/PubMed/PMC of the American National Library, American Chemical Abstracts (CA), Holland Medical Abstracts (EM), Western Pacific Region Index Medicus (WPRIM), Scopus and EBSCO. It is a monthly periodical published on the 15th of every month, and is distributed both at home and overseas. The Chinese series publication number is CN 43-1301/R；ISSN 1008-8830. The tenet of CJCP is to “reflect the latest advances and be open to the world”. The periodical reports the most recent advances in the contemporary pediatric field. The majority of the readership is pediatric doctors and researchers.