本文選題：轉(zhuǎn)甲狀腺素蛋白 + 淀粉樣變��； 參考：《國際藥學研究雜志》2017年02期

【摘要】：轉(zhuǎn)甲狀腺素蛋白(TTR)淀粉樣變是由于TTR沉積所致的系統(tǒng)性疾病,周圍神經(jīng)及心臟是主要受累臟器,嚴重影響患者的生活質(zhì)量和生命。目前的治療方法有限,迫切需要有新型療法延緩或者逆轉(zhuǎn)疾病進展。TTR穩(wěn)定藥物,如二氟尼柳和氯苯唑酸可延緩神經(jīng)病變的進展,但對心肌病變是否有效仍需進一步研究。清除已形成淀粉樣物質(zhì)的藥物(多西環(huán)素/�；切苋パ跄懰岷涂寡宓矸蹣游镔|(zhì)P抗體)目前在早期研發(fā)階段,一旦成功,將能用于任何類型的系統(tǒng)性淀粉樣變�；虺聊�(如反義寡核苷酸和小干擾RNA)技術(shù)目前正在進行Ⅱ/Ⅲ期臨床試驗,初步顯示能持續(xù)顯著抑制突變和野生TTR合成,可能會進一步逆轉(zhuǎn)淀粉樣變。
[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，

本文編號：1751131