期刊
JOURNAL OF NEUROSCIENCE
卷 38, 期 44, 页码 9375-9382出版社
SOC NEUROSCIENCE
DOI: 10.1523/JNEUROSCI.1666-18.2018
关键词
Parkinson's disease; alpha-synuclein; LRRK2; GBA; disease-modifying therapies
资金
- Roche
- Renovo
- Teva/Lundbeck
- Merck
- Biogen
The greatest unmet therapeutic need in Parkinson's disease (PD) is a treatment that slows the relentless progression of the symptoms and the neurodegenerative process. This review highlights the utility of genetics to understand the pathogenic mechanisms and develop novel therapeutic approaches for PD. The focus is on strategies provided by genetic studies: notably via the reduction and clearance of alpha-synuclein, inhibition of LRRK2 kinase activity, and modulation of glucocerebrosidase-related substrates. In addition, the critical role of precompetitive public-private partnerships in supporting trial design optimization, overall drug development, and regulatory approvals is illustrated. With these great advances, the promise of developing transformative therapies that halt or slow disease progression is a tangible goal.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据