Current and emerging treatment modalities for spinocerebellar ataxias.
Current and emerging treatment modalities for spinocerebellar ataxias.
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DOI：
10.1080/14737175.2022.2029703
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发表时间：
2022-03
影响因子：
4.3
通讯作者：
Zesiewicz, Theresa A.
中科院分区：
文献类型：
--
作者：
Ghanekar, Shaila D.;Kuo, Sheng-Han;Staffetti, Joseph S.;Zesiewicz, Theresa A.
脊髓小脑共济失调（SCA）是一组罕见的神经退行性疾病，严重影响受影响的个人及其家庭的生活。尽管对SCA的病因有了清楚的了解，但目前还没有FDA批准的对症或神经保护治疗。研究工作极大地扩展了潜在治疗的可能性，包括药物和非药物干预。基于基因治疗、神经刺激和分子靶向的新疗法也受到了极大的关注。这篇综述文章将讨论SCA治疗的当前进展以及即将出现的潜在干预措施。SCA是一个高度复杂和多方面的疾病家族，大多数研究强调对症药物治疗。由于SCA的临床前试验和其他神经退行性疾病的临床试验阐明了疾病修饰疗法（如AAV介导的基因疗法和ASO）的疗效，因此在症状前阶段解决SCA的潜力越来越有希望。
Spinocerebellar ataxias (SCA) are a group of rare neurodegenerative diseases that dramatically affect the lives of affected individuals and their families. Despite having a clear understanding of SCA’s etiology, there are no current symptomatic or neuroprotective treatments approved by the FDA. Research efforts have greatly expanded the possibilities for potential treatments, including both pharmacological and non-pharmacological interventions. Great attention is also being given to novel therapeutics based in gene therapy, neurostimulation, and molecular targeting. This review article will address the current advances in the treatment of SCA and what potential interventions are on the horizon. SCA is a highly complex and multifaceted disease family with the majority of research emphasizing symptomatic pharmacologic therapies. As pre-clinical trials for SCA and clinical trials for other neurodegenerative conditions illuminate the efficacy of disease modifying therapies such as AAV-mediated gene therapy and ASOs, the potential for addressing SCA at the pre-symptomatic stage is increasingly promising.
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影响因子：
2.5
作者：
Bushart DD;Shakkottai VG
通讯作者：
Shakkottai VG
影响因子：
5.7
作者：
Cho, Lily T. -Y.;Alexandrou, Aristos J.;Young, Gareth T.
通讯作者：
Young, Gareth T.
脊椎小脑共济失调：治疗开发的前景和挑战。
DOI：
10.1038/s41582-018-0051-6
发表时间：
2018-10
期刊：
Nature reviews. Neurology
影响因子：
--
作者：
Ashizawa T;Öz G;Paulson HL
通讯作者：
Paulson HL
影响因子：
2.5
作者：
Egorova, Polina A.;Zakharova, Olga A.;Bezprozvanny, Ilya B.
通讯作者：
Bezprozvanny, Ilya B.
影响因子：
11
作者：
Botez, MI;BotezMarquard, T;Lalonde, R
通讯作者：
Lalonde, R