Proteomic Data Advance Targeted Drug Development for Neurogenerative Diseases.

Proteomic Data Advance Targeted Drug Development for Neurogenerative Diseases.
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DOI:
10.1016/j.biopsych.2023.02.003
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发表时间:
2023-05-01
影响因子:
10.6
通讯作者:
--
中科院分区:
医学1区
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--
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神经退行性疾病,包括阿尔茨海默病、额颞叶痴呆、帕金森氏病、肌萎缩侧索硬化症(ALS)和多发性硬化症,是一组因神经元丧失而导致功能丧失的疾病。虽然这些疾病对个人和家庭来说都是沉重的负担,但仍然严重缺乏可用于治疗的治疗药物,因此有必要推动开发新药。不幸的是,药物开发仍然面临着高失败率,正如目前的阿尔茨海默病药物所看到的那样。研究人员继续推进研究神经退行性疾病药物开发的新途径。在本期的Ge等人(1)中,作者探索了大脑和血液的蛋白质组学数据,以寻找治疗方案的新见解。Ge等人强调,蛋白质对这些疾病中失去的一些功能至关重要,并且经常成为成功药物的靶点。大脑的蛋白质组可以解释病理,比如神经元的丢失,血液中的蛋白质能够穿过血脑屏障到达目标。Ge等人整理了一份他们认为与神经退行性疾病相关的22种蛋白质清单,如ACE、MAP1S、GRN和GPNMB;其中一些正在通过国家衰老研究所的阿尔茨海默病加速药物合作计划(AMP-AD)进行独立的药物开发。
Neurodegenerative diseases, including Alzheimer’s disease, Frontotemporal dementia, Parkinson’s disease, amyotrophic lateral sclerosis (ALS) and multiple sclerosis, are a group of diseases known for loss of neurons, leading to loss of function. While these diseases are onerous for both the individual and the family, there is still a severe lack of therapeutic drugs available for treatment, necessitating a push to develop novel drugs. Unfortunately, drug development continues to face a high failure rate, as seen with current Alzheimer’s drugs. Researchers continue to press forward to investigate new avenues of drug development for neurodegenerative disease.In Ge et al.(1), included in this issue, authors explore proteomic data from both the brain and blood to search for new insight into therapeutic options. Ge et al. highlight that proteins are critical to some of the functions lost in these diseases and are often targeted in successful drugs. The brain’s proteome can account for pathology, such as neuron loss, and blood proteins are able to cross the blood-brain barrier to reach targets. Ge et al. curated a list of 22 proteins that they believe to be relevant to neurodegenerative diseases, such as ACE, MAP1S, GRN, and GPNMB; several of these are being looked at independently for drug development with the Accelerating Medicines Partnership Program for Alzheimer’Disease (AMP-AD) through the National Institute on Aging.
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