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相关概念视频

Gene Therapy00:59

Gene Therapy

27.6K
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...
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Translation01:31

Translation

156.6K
Lesson: Translation
Translation is the process of synthesizing proteins from the genetic information carried by messenger RNA (mRNA). Following transcription, it constitutes the final step in the expression of genes. This process is carried out by ribosomes, complexes of protein and specialized RNA molecules. Ribosomes, transfer RNA (tRNA), and other proteins produce a chain of amino acids—the polypeptide—as the end product of translation.
Translation Produces the Building Blocks of...
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Translation01:31

Translation

17.8K
Translation is the process of synthesizing proteins from the genetic information carried by messenger RNA (mRNA). Following transcription, it constitutes the final step in the expression of genes. This process is carried out by ribosomes, complexes of protein and specialized RNA molecules. Ribosomes, transfer RNA (tRNA), and other proteins produce a chain of amino acids—the polypeptide—as the end product of translation.
Translation Produces the Building Blocks of Life
Proteins are...
17.8K
Initiation of Translation02:33

Initiation of Translation

39.0K
Initiating translation is complex because it involves multiple molecules. Initiator tRNA, ribosomal subunits, and eukaryotic initiation factors (eIFs) are all required to assemble on the initiation codon of mRNA. This process consists of several steps that are mediated by different eIFs.
First, the initiator tRNA must be selected from the pool of elongator tRNAs by eukaryotic initiation factor 2 (eIF2). The initiator tRNA (Met-tRNAi) has conserved sequence elements including modified bases at...
39.0K
Improving Translational Accuracy02:07

Improving Translational Accuracy

14.9K
Base complementarity between the three base pairs of mRNA codon and the tRNA anticodon is not a failsafe mechanism. Inaccuracies can range from a single mismatch to no correct base pairing at all. The free energy difference between the correct and nearly correct base pairs can be as small as 3 kcal/ mol. With complementarity being the only proofreading step, the estimated error frequency would be one wrong amino acid in every 100 amino acids incorporated. However, error frequencies observed in...
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Termination of Translation01:44

Termination of Translation

27.7K
The large ribosomal subunit has several important structures essential to translation. These include the peptidyl transferase center (PTC) - which is the site where the peptide bond is formed - and a large, internal, water-filled tube through which the nascent polypeptide moves. This latter structure is called the Peptide Exit Tunnel, and it begins at the PTC and spans the body of the large ribosomal subunit. During translation, as the nascent polypeptide chain is synthesized, it passes through...
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相关实验视频

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Determination of the Photoisomerization Quantum Yield of a Hydrazone Photoswitch
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基于光开关技术的内耳基因治疗的不太侵入性光照射方法:向临床转化方向

Masao Noda1,2, Takahiro Otabe3,4, Ryota Koshu1

  • 1Department of Otolaryngology and Head and Neck Surgery, Jichi Medical University, Tochigi, Japan.

Human gene therapy
|February 4, 2026
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概括

这项研究表明,使用光激活的Cre复合酶 (PA-Cre) 治疗小鼠听力损失的新型基因治疗方法. 外耳道和耳膜辐射有效地激活了耳中的基因表达,为内耳基因疗法提供了最少的侵入性方法.

关键词:
在 AAV AAV AAV 中.基因治疗的基因疗法内部的毛细胞是毛细胞.光照射辐射的光照射.照片交换机可以切换照片.

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科学领域:

  • 耳鼻喉科 耳鼻喉科 耳鼻喉科
  • 基因治疗 基因治疗
  • 分子生物学分子生物学

背景情况:

  • 听力障碍是痴呆症的重要风险因素,目前的治疗方法,如助听器,提供有限的益处.
  • 内耳基因疗法为治疗感觉神经听力损失提供了一个有前途的途径,但需要可控制和最少侵入的基因激活方法.
  • 一个可光激活的Cre复合酶 (PA-Cre) 系统以前被开发用于空间时间基因表达调节.

研究的目的:

  • 评估使用外部听道 (EAC) 和耳膜 (TM) 辐射用于最小侵入性激活耳基因表达的可行性.
  • 评估通过腺相关病毒载体 (AAV.GTX) 传递的PA-Cre系统在小鼠内耳中通过光介导的基因激活的疗效.
  • 为了比较不同的光照射方法 (直耳,TM和EAC) 在耳中的基因重组效率.

主要方法:

  • 编码PA-Cre的腺相关病毒载体 (AAV.GTX) 和一个Cre依赖的记者被通过圆窗膜注入C57BL/6J小鼠的耳.
  • 注射后七天,通过三种方法进行光照射:直接进入耳,TM光纤探针和非侵入性EAC照射.
  • 在内部毛细胞 (IHCs) 中的重组效率是使用全免疫组织化学量化,测量记者基因表达 (tdTomato).

主要成果:

  • AAV.GTX有效地转化了IHC,并显示了低的基底CRE活性 (<5% tdTomato表达没有光).
  • 直接耳辐射实现了高的重组率 (88.4 ± 1.5%).
  • TM和EAC照射导致可比且高的转换效率 (分别为95.8±1.7%和97.6±1.2%),而不会损害耳完整性.

结论:

  • 在小鼠耳中,PA-Cre系统有效地运行,背景活动最小.
  • 最少侵入性TM和EAC辐射能够在内耳中强有力的和可控的光介导基因激活.
  • 这一策略为开发用于治疗听力损失和其他耳疾病的非侵入性,光控制基因治疗提供了基础.