相关实验视频
Updated: Jul 27, 2026

11:49
Trans-vivo Delayed Type Hypersensitivity Assay for Antigen Specific Regulation
Published on: May 2, 2013
在长期酶替代疗法后的免疫耐受性,在患有粘多糖症I的患者中
Revecca Kakavanos1, Chris T Turner, John J Hopwood
1Lysosomal Diseases Research Unit, Department of Chemical Pathology, Women's and Children's Hospital, North Adelaide, South Australia, Australia
Lancet (London, England)
|May 16, 2003
概括
接受酶替代疗法的I型粘多糖症患者在两年内对alpha-L-iduronidase产生免疫耐受性. 这表明这种罕见的遗传疾病具有积极的长期治疗潜力.
科学领域:
- 免疫学 免疫学 免疫学
- 遗传学 是一个遗传学.
- 生物化学 生物化学
背景情况:
- 粘多糖症I (MPS I) 是一种罕见的遗传疾病,由α-L-氨基酶缺乏引起.
- 使用复合人体α-L-氨基酶的酶替代疗法 (ERT) 是MPS I的治疗选择.
- 研究对ERT的免疫反应对于长期治疗疗效至关重要.
研究的目的:
- 在接受ERT的MPS I患者中研究对复合人体α-L-氨基酶的幽默免疫反应.
- 在104周的治疗期内表征抗体标位和表皮质反应性.
主要方法:
- 从十名MPS I患者的血清样本在基线和多个时间点分析到104周.
- 测量了抗体标位和对alpha-L-iduronidase的特定线性序列表皮位反应性.
- 患者样本值与正常人对照值进行了比较.
主要成果:
- 最初,所有患者的抗体标位都在正常范围内较低.
- 五名患者在治疗期间出现了高于正常的抗体标位和高亲缘关系表皮质反应活性.
- 到第104周,所有患者的抗体标位和低亲和性表皮质反应活性都较低,这表明免疫反应降低.
结论:
- 最初获得免疫反应的患者在两年ERT后对alpha-L-iduronidase产生了免疫耐受性.
- 免疫耐受性的这种发展对MPS I患者ERT的长期安全性和有效性产生了积极的影响.
- 在6-12周的早期正常抗体标位与没有随后免疫反应的发展相关.
相关概念视频
Cell-mediated Immune Responses
Overview
Lysosomal Hydrolases
Lysosomes are the site for the degradation of macromolecules and biological polymers released during membrane trafficking events such as secretory, endocytic, autophagic, and phagocytic pathways. The membrane-enclosed area of the lysosome, called the lumen, contains hydrolytic enzymes active in an acidic environment. These acid hydrolases are functional at a pH between 4.5 and 5 and are involved in cellular processes such as cell signaling, energy metabolism, restoration of the plasma membrane,...
Tissue Transplantation
Tissue transplantation is a significant medical procedure involving the transfer of cells, tissues, or organs from a donor to a recipient, with the primary aim of restoring lost functions. This procedure is crucial in treating a broad spectrum of diseases, including kidney diseases, liver failure, heart disease, and certain types of cancers.
The Biology of Tissue Transplantation
The biology of tissue transplantation hinges on the Major Histocompatibility Complex (MHC) molecules. These molecules...
The Biology of Tissue Transplantation
The biology of tissue transplantation hinges on the Major Histocompatibility Complex (MHC) molecules. These molecules...
Pharmacogenetics of Phase II Enzymes: N-acetyltransferase, Thiopurine S-methyltransferase, UDP-glucuronosyltransferase
Phase II biotransformation reactions are essential for detoxifying and eliminating xenobiotics, including many pharmaceutical compounds. These reactions typically involve conjugation, the covalent attachment of polar endogenous groups such as glucuronic acid, sulfate, methyl, or acetyl moieties to functional groups introduced during Phase I metabolism. The resulting conjugates are more water-soluble, enabling efficient renal or biliary excretion.The major classes of Phase II enzymes include...

