转甲状腺素蛋白淀粉样变药物治疗研究进展

发布时间：2018-04-14 21:44

  本文选题：转甲状腺素蛋白 + 淀粉样变　； 参考：《国际药学研究杂志》2017年02期

【摘要】：转甲状腺素蛋白(TTR)淀粉样变是由于TTR沉积所致的系统性疾病,周围神经及心脏是主要受累脏器,严重影响患者的生活质量和生命。目前的治疗方法有限,迫切需要有新型疗法延缓或者逆转疾病进展。TTR稳定药物,如二氟尼柳和氯苯唑酸可延缓神经病变的进展,但对心肌病变是否有效仍需进一步研究。清除已形成淀粉样物质的药物(多西环素/牛磺熊去氧胆酸和抗血清淀粉样物质P抗体)目前在早期研发阶段,一旦成功,将能用于任何类型的系统性淀粉样变。基因沉默(如反义寡核苷酸和小干扰RNA)技术目前正在进行Ⅱ/Ⅲ期临床试验,初步显示能持续显著抑制突变和野生TTR合成,可能会进一步逆转淀粉样变。
[Abstract]:TTR amyloidosis is a systemic disease caused by the deposition of TTR. Peripheral nerve and heart are the main involved organs, which seriously affect the quality of life and life of patients.The current treatment methods are limited and there is an urgent need for new therapies to delay or reverse the progress of the disease. TTR stabilizers such as difluniline and chlorobenzoic acid can delay the progression of neuropathy but the effectiveness of cardiomyopathy remains to be further studied.Drugs (doxycycline / taurodeoxycholic acid and anti-serum amyloid P antibody) that have formed amyloid are currently in the early stages of development and, if successful, will be used in any type of systemic amyloidosis.Gene silencing (such as antisense oligodeoxynucleotides and small interfering RNAs) is currently undergoing phase II / 鈪，

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