口腔耐受在基因治疗中的潜在作用。

IF 3.7 4区 医学 Q2 CELL BIOLOGY Cellular immunology Pub Date : 2023-09-01 DOI:10.1016/j.cellimm.2023.104742
John S.S. Butterfield , Xin Li , Sreevani Arisa , Kwang-Chul Kwon , Henry Daniell , Roland W. Herzog
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引用次数: 2

摘要

目前正在开发针对各种自身免疫性疾病和过敏的口服免疫疗法,以抗原特异性的方式抑制免疫反应。先前的研究表明,在遗传性出血性疾病血友病的蛋白质替代治疗中,通过反复口服生物胶囊化的凝血因子抗原,可以防止抗药物抗体(抑制剂)的形成。在这里,我们发现这种方法显著减少了接受腺相关病毒基因转移治疗的血友病A小鼠中针对因子VIII的抗体产生。我们提出,口服耐受的概念可以应用于预防针对基因治疗中表达的治疗性转基因产物的免疫反应。
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Potential role for oral tolerance in gene therapy

Oral immunotherapies are being developed for various autoimmune diseases and allergies to suppress immune responses in an antigen-specific manner. Previous studies have shown that anti-drug antibody (inhibitor) formation in protein replacement therapy for the inherited bleeding disorder hemophilia can be prevented by repeated oral delivery of coagulation factor antigens bioencapsulated in transplastomic lettuce cells. Here, we find that this approach substantially reduces antibody development against factor VIII in hemophilia A mice treated with adeno-associated viral gene transfer. We propose that the concept of oral tolerance can be applied to prevent immune responses against therapeutic transgene products expressed in gene therapy.

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来源期刊
Cellular immunology
Cellular immunology 生物-免疫学
CiteScore
8.20
自引率
2.30%
发文量
102
审稿时长
30 days
期刊介绍: Cellular Immunology publishes original investigations concerned with the immunological activities of cells in experimental or clinical situations. The scope of the journal encompasses the broad area of in vitro and in vivo studies of cellular immune responses. Purely clinical descriptive studies are not considered. Research Areas include: • Antigen receptor sites • Autoimmunity • Delayed-type hypersensitivity or cellular immunity • Immunologic deficiency states and their reconstitution • Immunologic surveillance and tumor immunity • Immunomodulation • Immunotherapy • Lymphokines and cytokines • Nonantibody immunity • Parasite immunology • Resistance to intracellular microbial and viral infection • Thymus and lymphocyte immunobiology • Transplantation immunology • Tumor immunity.
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