基因疗法:治疗阿尔茨海默病的替代方法
Vanshika Doshi1, Garima Joshi2, Sanjay Sharma3
1Shobhaben Pratapbhai Patel School of Pharmacy & Technology Management, SVKM's NMIMS (Deemed to be University), Vile Parle West, Mumbai, Maharashtra, 400056, India.
Naunyn-Schmiedeberg's archives of pharmacology
|December 11, 2023
概括
基因疗法为阿尔茨海默病 (AD) 提供了一种有希望的疾病修饰方法,阿尔茨海默病是一种神经退行性疾病. 这种创新治疗涉及将基因传递到目标大脑部位,可能改变AD的进展.
科学领域:
- 神经科学是一个神经科学.
- 遗传学 是一个遗传学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 阿尔茨海默病 (AD) 是一种进展性神经退行性疾病,其特点是记忆力丧失和认知能力下降.
- 目前的AD治疗提供症状缓解,但不会改变疾病病理生理学.
- 对阿尔茨海默病的关键假设包括粉样β斑块的积累和神经纤维状结 (NFT) 的形成.
研究的目的:
- 探索基因疗法作为阿尔茨海默病潜在的疾病修饰治疗方法.
- 讨论AD的基因传递方法的机制,优势和局限性.
- 审查AD的病理生理学,并确定基因治疗的治疗点.
主要方法:
- 对有关阿尔茨海默病病理生理学和治疗假设的现有文献的综述.
- 对基因治疗原理的分析,包括病毒和非病毒载体传递系统.
- 在阿尔茨海默氏症疾病级联中识别潜在的基因标.
主要成果:
- 基因疗法为阿尔茨海默病的疾病修饰提供了一种新的策略.
- 不同的基因传递载体 (病毒和非病毒) 对阿兹海默病治疗有不同的特征.
- 在AD病理生理学中,多个分子点容易接受基因治疗干预.
结论:
- 基因疗法作为阿尔茨海默病的疾病修饰治疗具有显著的潜力.
- 了解AD病理生理学对于优化基因疗法标和输送至关重要.
- 对基因传递系统和治疗点进行进一步的研究是有效的AD基因治疗的必要条件.
相关概念视频
01:22Alzheimer's Disease: Treatment
195
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...
195
01:26Alzheimer's Disease: Overview
492
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...
492
00:59Gene Therapy
25.4K
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...
25.4K
02:30siRNA - Small Interfering RNAs
16.8K
Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
16.8K
01:30Cognitive Enhancers: Cholinesterase Inhibitors and NMDA Receptor Antagonists
128
Cognitive enhancers, also known as "smart drugs," are substances used to enhance memory, mental alertness, and concentration. These can be natural or synthetic and improve cognition in conditions like Alzheimer's disease (AD) and other neurodegenerative diseases. Some common examples include caffeine, amphetamines, methylphenidate, modafinil, arecoline, donepezil, vortioxetine, and piracetam. These enhancers work on the principle of synaptic plasticity and altered circuit function.
128