CTAD专题组:阿尔茨海默病的遗传疗法。

IF 4.3 Q2 BUSINESS
D Jakabek, A M Isaacs, B De Strooper, M Tuszynski, R Lane, O Uspenskaya, E McDade, M S Rafii, C J Mummery
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引用次数: 0

摘要

治疗阿尔茨海默病和其他痴呆症的遗传方法越来越多,在早期试验中取得了一些有希望的结果。这促使欧盟-美国在2024年10月召开了第一个关于阿尔茨海默病遗传疗法的CTAD特别工作组会议。基因疗法在阿尔茨海默病和其他痴呆症的临床前研究和临床试验在这里介绍了该领域的关键经验教训。重要的是,我们回顾和讨论了神经遗传疗法所特有的一些挑战和机遇,包括基因操作手段、不良事件、监测、治疗时机以及患者参与试验设计的重要性。跨学科的持续合作将加速神经遗传疗法的发展。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
CTAD taskforce: genetic therapies in Alzheimer's disease.

There are an increasing number of genetic approaches to treating Alzheimer's disease and other dementias, with some promising results from early-phase trials. This prompted the convention of the first EU-US CTAD Task Force on genetic therapies in Alzheimer's disease in October 2024. Preclinical studies and clinical trials of genetic therapies in Alzheimer's disease and other dementias are presented here with key lessons for the field. Importantly, there are several challenges and opportunities unique to neurogenetic therapies which were reviewed and discussed, including means of genetic manipulation, adverse events, monitoring, timing of therapy, and the importance of patient involvement in trial design. Continued collaboration across disciplines will accelerate development of neurogenetic therapeutics.

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来源期刊
The Journal of Prevention of Alzheimer's Disease
The Journal of Prevention of Alzheimer's Disease Medicine-Psychiatry and Mental Health
CiteScore
9.20
自引率
0.00%
发文量
0
期刊介绍: The JPAD Journal of Prevention of Alzheimer’Disease will publish reviews, original research articles and short reports to improve our knowledge in the field of Alzheimer prevention including: neurosciences, biomarkers, imaging, epidemiology, public health, physical cognitive exercise, nutrition, risk and protective factors, drug development, trials design, and heath economic outcomes.JPAD will publish also the meeting abstracts from Clinical Trial on Alzheimer Disease (CTAD) and will be distributed both in paper and online version worldwide.We hope that JPAD with your contribution will play a role in the development of Alzheimer prevention.
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