基因突变引起的神经退行性疾病研究性治疗: 近期临床试验经验

BP Imbimbo, V Triaca, C Imbimbo… - 中国神经再生研究(英文版), 2023 - sjzsyj.com.cn
We reviewed recent major clinical trials with investigational drugs for the treatment of
subjects with neurodegenerative diseases caused by inheritance of gene mutations or …

Investigational treatments for neurodegenerative diseases caused by inheritance of gene mutations: lessons from recent clinical trials

BP Imbimbo, V Triaca, C Imbimbo… - Neural Regeneration …, 2023 - journals.lww.com
We reviewed recent major clinical trials with investigational drugs for the treatment of
subjects with neurodegenerative diseases caused by inheritance of gene mutations or …

New hope for therapy in neurodegenerative diseases

M do Carmo Costa, HL Paulson - Cell Research, 2013 - nature.com
The use of genetic screens to define cellular pathways that regulate neurodegenerative
disease proteins has emerged as a powerful strategy to identify potential therapeutic targets …

神经退行性疾病病变机理及药物作用新靶点

朱兴族 - 世界科技研究与发展, 1999 - cqvip.com
神经退行性疾病是医学研究领域中的一大难题, 近年来, 该类疾病遗传因素及病变机理的研究
取得了很大进展. 研究表明这类疾病的发生与某些基因突变后产生的蛋白有关 …

The future of genetic research on neurodegeneration

C Van Broeckhoven - Nature medicine, 2010 - nature.com
Why, with all the progress in the field of neurodegeneration, do we still lack disease-
modifying drugs that tackle the primary defect of severe cell loss? How much progress has …

Gene-based therapies for neurodegenerative diseases

J Sun, S Roy - Nature neuroscience, 2021 - nature.com
Gene therapy is making a comeback. With its twin promise of targeting disease etiology and
'long-term correction', gene-based therapies (defined here as all forms of genome …

[HTML][HTML] Application of gene editing in neurodegenerative diseases, volume II

F Qi, X Xu, X Guo, J Luo, Y Li, S Yan - Frontiers in Neuroscience, 2023 - frontiersin.org
Neurodegenerative disorders (NDs) are a broad category of ailments caused by progressive
damage to cells and the nervous system that affect millions of individuals worldwide. The …

[HTML][HTML] Gene therapy for neurodegenerative disease: clinical potential and directions

X Zhu, Y Zhang, X Yang, C Hao… - Frontiers in Molecular …, 2021 - frontiersin.org
The pathogenesis of neurodegenerative diseases (NDDs) is complex and diverse. Over the
decades, our understanding of NDD has been limited to pathological features. However …

The role of gene editing in neurodegenerative diseases

HC Fan, CS Chi, YJ Lee, JD Tsai, SZ Lin… - Cell …, 2018 - journals.sagepub.com
Neurodegenerative diseases (NDs), at least including Alzheimer's, Huntington's, and
Parkinson's diseases, have become the most dreaded maladies because there are no …

Gene therapy and pharmacological treatment of inherited neurological disorders

JB Martin - Trends in biotechnology, 1995 - cell.com
The identification of mutant genes responsible for inherited neurological disorders gives an
opportunity to consider new approaches to their treatment. Although replacement of …