对于阿尔茨海默病的生长因子基因治疗
1Department of Neurosciences, University of California San Diego, La Jolla, CA, USA.
Journal of Alzheimer's disease : JAD
|October 18, 2024
概括
这项研究调查了使用基因疗法为阿尔茨海默氏症提供脑衍生神经营养因子 (BDNF) 的研究.
科学领域:
- 神经科学是一个神经科学.
- 遗传学 是一个遗传学.
- 神经退行性疾病 神经退行性疾病
背景情况:
- 神经系统生长因子,如来自大脑的神经营养因子 (BDNF),对于神经元的生存和功能至关重要.
- 在阿尔茨海默病 (AD) 动物模型中,BDNF表现出神经保护作用,增强突触可塑性,改善认知功能.
- 之前在AD患者中NGF基因疗法试验证实,生长因子可以引起退化的神经元中的热量反应.
研究的目的:
- 评估BDNF的基因传递在减缓阿尔茨海默氏病进展方面的有效性.
- 为了确定BDNF基因疗法是否可以改善AD患者的记忆和认知功能.
主要方法:
- 基因输送的BDNF正在被用于诊断为阿尔茨海默病的患者.
- 目前正在进行临床试验NCT05040217 (2021年6月开始).
- 从之前的神经生长因子 (NGF) 基因治疗试验 (NCT00017940,2001年6月) 的经验中获益,以优化BDNF输送.
主要成果:
- 摘要中没有包含当前试验的具体结果,因为它正在进行中.
- 以前的NGF基因疗法试验表明,生长因子可以刺激AD的神经元修复.
结论:
- 作为阿尔茨海默病的治疗策略,BDNF的基因传递具有前景.
- 优化基因传递方法是最大限度地提高神经退行性疾病中生长因子的治疗潜力的关键.
相关概念视频
Gene Therapy
25.3K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
25.3K
Alzheimer's Disease: Treatment
165
Alzheimer's Disease (AD), a neurodegenerative disorder, is pathologically identified by amyloid plaques and neurofibrillary tangles composed of tau protein. AD pharmacotherapy aims to manage cognitive symptoms, delay disease progression, and treat behavioral symptoms. The treatment is primarily symptomatic and palliative, with no definitive disease-modifying therapy available. Cholinesterase inhibitors, including donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), are...
165
iPS Cell Differentiation
2.6K
The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
2.6K
Alzheimer's Disease: Overview
449
Alzheimer's Disease (AD) is a continually advancing neurodegenerative disorder, distinguished by escalating memory loss, cognitive dysfunction, and dementia. The disease unfolds in three stages: preclinical, mild cognitive impairment (MCI), and dementia. Its onset is insidious, and the progression gradual, with the cause not well explained by other disorders.
The clinical diagnosis of AD hinges on the presence of memory and other cognitive impairments. Biomarkers, such as changes in Aβ...
The clinical diagnosis of AD hinges on the presence of memory and other cognitive impairments. Biomarkers, such as changes in Aβ...
449


