相关实验视频
Updated: Jul 26, 2025
13:10
DNA Vector-based RNA Interference to Study Gene Function in Cancer
Published on: June 4, 2012
20.6K
针对各种疾病的基于RNA的小干扰基因药物的出现
Harshini Kurakula1, Swetha Vaishnavi1, Mohammed Yaseen Sharif1
1Department of Chemistry, School of Engineering and Sciences, SRM University-AP, Amaravati, Andhra Pradesh 522240, India.
ACS omega
|June 16, 2023
概括
小干扰RNA (siRNA) 基因疗法为各种疾病的传统药物提供了有希望的替代品. 必须克服分娩,稳定性和免疫反应方面的挑战,才能充分发挥其治疗潜力.
科学领域:
- 生物技术是生物技术.
- 遗传学 遗传学 是一个
- 药理学 药理学是指药理学的学科.
背景情况:
- 基因疗法正在成为传统的小分子,和基于蛋白质的药物的重要替代品.
- 发现RNA干扰 (RNAi) 加快了基于siRNA的基因疗法的发展.
- 几种FDA批准的siRNA药物证明了这种治疗方式的日益增长的潜力.
研究的目的:
- 提供基于siRNA的基因药物的全面概述.
- 讨论siRNA传递系统的挑战和潜力.
- 探索基于siRNA的治疗方法的未来前景.
主要方法:
- 对基于siRNA的基因治疗现有文献的审查.
- 对FDA批准的siRNA药物及其应用的分析.
- 确定siRNA传递和有效性的关键挑战.
主要成果:
- 基于siRNA的疗法在治疗诸如遗传性跨氨酸中介性粉症和急性肝脏病等疾病方面取得了成功.
- siRNA药物比其他基因疗法具有优势,并且正在针对各种疾病进行研究.
- 关键的挑战包括化学不稳定性,生物分布,免疫反应和非目标效应.
结论:
- 基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因基因
- 克服交付和安全挑战对于广泛的临床采用至关重要.
- 需要进一步的研究和开发,才能充分利用siRNA技术的能力.
相关概念视频
02:30siRNA - Small Interfering RNAs
16.9K
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...
16.9K
02:15Experimental RNAi
6.2K
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
6.2K
02:30Small interfering RNAs (siRNA)
3.6K
3.6K
00:59Gene Therapy
25.6K
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...
25.6K
01:22MicroRNAs
3.0K
MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...
3.0K
01:29Microorganisms in Medicine and Therapeutics
72
Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
72