Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.

IF 4.7 4区医学 Q1 OBSTETRICS & GYNECOLOGY

Obstetrical & Gynecological Survey Pub Date : 2025-11-01 DOI:10.1097/OGX.0000000000001462

Claire Booth, Julián Sevilla, Elena Almarza, Caroline Y Kuo, Josune Zubicaray, Dayna Terrazas, Gráinne O'Toole, Maria Chitty-Lopez, Grace Choi, Eileen Nicoletti, Janel Long-Boyle, Augustine Fernandes, Kritika Chetty, Satiro De Oliveira, Crystal Banuelos, Jinhua Xu-Bayford, Antonella Lucía Bastone, Philipp John-Neek, Connie Jackson, Theodore B Moore, Kimberly Gilmour, Axel Schambach, Michael Rothe, Sanchali Kasbekar, Gayatri R Rao, Kinnari Patel, Gaurav Shah, Adrian J Thrasher, Juan A Bueren, Jonathan D Schwartz, Donald B Kohn

引用次数: 0

Abstract

(Abstracted from N Engl J Med 2025;392:1698-1709) Leukocyte adhesion deficiency type I (LAD-I) is an autosomal recessive immune disorder marked by recurrent, life-threatening infections, with 25% to 39% mortality before age 2 years without curative therapy. Common symptoms include umbilical-cord complications, poorly healing cutaneous lesions, persistent leukocytosis, and periodontitis.

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慢病毒基因治疗严重白细胞粘附缺陷1型。

（摘要自中华医学杂志2025；392:1698-1709）白细胞粘附缺乏症I型（LAD-I）是一种常染色体隐性免疫疾病，以复发性、危及生命的感染为特征，2岁前未经根治性治疗的死亡率为25%至39%。常见症状包括脐带并发症、愈合不良的皮肤病变、持续的白细胞增多和牙周炎。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Obstetrical & Gynecological Survey 医学-妇产科学

CiteScore

2.70

自引率

3.20%

发文量

245

审稿时长

>12 weeks

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