New insights into the therapeutic approaches for the treatment of tauopathies.
New insights into the therapeutic approaches for the treatment of tauopathies.
复制标题
对tau蛋白病治疗方法的新见解。
DOI：
10.4103/1673-5374.385288
复制
发表时间：
2024-05
影响因子：
6.1
通讯作者：
Pourmotabbed T
中科院分区：
文献类型：
--
作者：
Singh H;Das A;Khan MM;Pourmotabbed T
tau蛋白病是一组神经系统疾病，包括阿尔茨海默病和额颞叶痴呆，其涉及进行性神经变性、认知缺陷和异常tau蛋白积累。目前不能通过治疗措施阻止或减缓tau蛋白病的发展。鉴于tau负荷在原发性tau蛋白病中的显著贡献以及致病性tau积累与认知缺陷之间的强关联，人们对创建可以减轻tau病理学并产生神经保护作用的疗法非常感兴趣。最近，小分子、免疫疗法和基因疗法已被用于减少病理性tau负担并预防tau蛋白病动物模型中的神经变性。然而，目前治疗方法的主要缺陷是药物和基因靶向方式难以穿过血脑屏障及其意外的副作用。在这篇综述中，目前用于tau蛋白病的治疗策略，包括使用基于阿糖胞苷的基因治疗方法，在临床前动物模型中治疗tau蛋白病和阿尔茨海默病，已显示出有希望的结果进行了讨论。
Tauopathies are a group of neurological disorders, including Alzheimer’s disease and frontotemporal dementia, which involve progressive neurodegeneration, cognitive deficits, and aberrant tau protein accumulation. The development of tauopathies cannot currently be stopped or slowed down by treatment measures. Given the significant contribution of tau burden in primary tauopathies and the strong association between pathogenic tau accumulation and cognitive deficits, there has been a lot of interest in creating therapies that can alleviate tau pathology and render neuroprotective effects. Recently, small molecules, immunotherapies, and gene therapy have been used to reduce the pathological tau burden and prevent neurodegeneration in animal models of tauopathies. However, the major pitfall of the current therapeutic approach is the difficulty of drugs and gene-targeting modalities to cross the blood-brain barrier and their unintended side effects. In this review, the current therapeutic strategies used for tauopathies including the use of oligonucleotide-based gene therapy approaches that have shown a promising result for the treatment of tauopathies and Alzheimer’s disease in preclinical animal models, have been discussed.
登录
查看更多内容
影响因子：
14.9
作者：
Anderson BA;Freestone GC;Low A;De-Hoyos CL;Iii WJD;Østergaard ME;Migawa MT;Fazio M;Wan WB;Berdeja A;Scandalis E;Burel SA;Vickers TA;Crooke ST;Swayze EE;Liang X;Seth PP
通讯作者：
Seth PP
影响因子：
5.3
作者：
DeVos, Sarah L.;Goncharoff, Dustin K.;Miller, Timothy M.
通讯作者：
Miller, Timothy M.
DOI：
10.1073/pnas.94.1.298
发表时间：
1997-01-07
影响因子：
11.1
作者：
Alonso, AD;GrundkeIqbal, I;Iqbal, K
通讯作者：
Iqbal, K
影响因子：
14
作者：
Borroni, Barbara;Graff, Caroline;Hardiman, Orla;Ludolph, Albert C.;Moreno, Fermin;Otto, Markus;Piccininni, Marco;Remes, Anne M.;Rowe, James B.;Seelaar, Harro;Stefanova, Elka;Traykov, Latchezar;Logroscino, Giancarlo
通讯作者：
Logroscino, Giancarlo
影响因子：
168.9
作者：
Cho, Eun-Ae;Moloney, Fergal J.;Cai, Hong;Au-Yeung, Annie;China, Carlos;Scolyer, Richard A.;Yosufi, Benafsha;Raftery, Mark J.;Deng, Jason Z.;Morton, Stephen W.;Hammond, Paula T.;Arkenau, Hendrik-Tobias;Damian, Diona L.;Francis, Douglas J.;Chesterman, Colin N.;Barnetson, Ross St C.;Halliday, Gary M.;Khachigian, Levon M.
通讯作者：
Khachigian, Levon M.