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相关概念视频

siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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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...
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Mesenchymal Stem Cells01:19

Mesenchymal Stem Cells

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Mesenchymal stem cells (MSCs) are adult stem cells that can differentiate into most connective tissue cell types, except for hematopoietic cells, depending upon the source of MSCs. For example, bone-marrow-derived MSCs (BM-MSCs) can differentiate into osteocytes, hepatocytes, and pancreatic and neuronal cells. MSCs can be isolated from various sources such as bone marrow, placenta, adipose tissue, teeth, and Wharton’s jelly, a gelatinous substance in the umbilical cord. The ease of their...
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MicroRNAs01:22

MicroRNAs

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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...
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相关实验视频

Updated: Jun 13, 2025

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
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在多发性硬化症中基于MiRNA的治疗潜力.

Ana Zabalza1, Agustin Pappolla1, Manuel Comabella1

  • 1Vall Hebron University Hospital & Research Institute (VHIR), Multiple Sclerosis Centre of Catalonia (Cemcat) & Neurology Department, Universitat Autonoma de Barcelona, Barcelona, Spain.

Frontiers in immunology
|September 13, 2024
PubMed
概括

微RNAs (miRNAs) 是多发性硬化症 (MS) 病原和结果的关键参与者. 本综述探讨了它们的诊断潜力和MS的治疗应用.

关键词:
生物标志物 生物标志物微RNAs 是一个微型RNA.多发性硬化症多发性硬化症神经学 神经学 神经学治疗目标是治疗的目标.

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Characterization of Functionally Associated miRNAs in Glioblastoma and their Engineering into Artificial Clusters for Gene Therapy
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A Simple Alternative to Stereotactic Injection for Brain Specific Knockdown of miRNA

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相关实验视频

Last Updated: Jun 13, 2025

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
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Characterization of Functionally Associated miRNAs in Glioblastoma and their Engineering into Artificial Clusters for Gene Therapy
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科学领域:

  • 神经科学是一个神经科学.
  • 分子生物学分子生物学
  • 遗传学 遗传学 是一个

背景情况:

  • 多发性硬化症 (MS) 是一种慢性自身免疫性疾病,影响中枢神经系统.
  • 目前对MS病理生理学,生物标志物和治疗方法的理解正在不断发展.
  • 微RNA (miRNA) 是小的非编码RNA,在基因表达中起着重要的调节作用.

研究的目的:

  • 审查微RNAs (miRNAs) 在多发性硬化症 (MS) 和其临床结果中的作用.
  • 探索miRNAs作为MS诊断和预后生物标志物的潜力.
  • 讨论目前和新兴的基于miRNA的MS治疗策略.

主要方法:

  • 在MS中调查miRNA的研究的文献综述.
  • 在生物流体 (血清,血,脑脊液) 中分析miRNA表达.
  • 基于miRNA的治疗方法的概述,包括恢复和抑制.

主要成果:

  • 特定的miRNAs在MS的各种临床形式中被差异地表达.
  • 在MS中,miRNA显示出作为诊断和预后生物标志物的潜力.
  • 目前正在开发各种基于miRNA的治疗策略来治疗MS.

结论:

  • miRNAs代表了多发性硬化症 (MS) 的有希望的治疗标和生物标志物.
  • 需要进一步的研究来验证治疗疗效,并优化基于miRNA的治疗的输送方法.
  • 解决交付和验证方面的局限性对于推进MS的miRNA疗法至关重要.