医学
免疫学
造血干细胞移植
调节性T细胞
疾病
移植
遗传增强
造血
基因
干细胞
T细胞
免疫系统
白细胞介素2受体
遗传学
内科学
生物
作者
Holly Wobma,Erin Janssen
标识
DOI:10.1016/j.rdc.2023.06.009
摘要
Regulatory T cells (Tregs) are critical for enforcing peripheral tolerance. Monogenic "Tregopathies" affecting Treg development, stability, and/or function commonly present with polyautoimmunity, atopic disease, and infection. While autoimmune manifestations may present in early childhood, as more disorders are characterized, conditions with later onset have been identified. Treg numbers in the blood may be decreased in Tregopathies, but this is not always the case, and genetic testing should be pursued when there is high clinical suspicion. Currently, hematopoietic cell transplantation is the only curative treatment, but gene therapies are in development, and small molecule inhibitors/biologics may also be used.
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