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相关概念视频

CRISPR01:59

CRISPR

49.1K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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CRISPR and crRNAs02:53

CRISPR and crRNAs

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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
16.6K

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从长椅到床边:开发基于CRISPR/Cas的治疗方法来治疗眼部疾病.

Qing Zhao1, Linxin Wei1, Youxin Chen1

  • 1Department of Ophthalmology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing 100730, China; Key Laboratory of Ocular Fundus Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College Hospital, Beijing 100730, China; Beijing Key Laboratory of Fundus Diseases Intelligent Diagnosis & Drug/Device Development and Translation, Beijing 100730, China.

Pharmacological research
|January 31, 2025
PubMed
概括

集群定期间隔的简短巴林德罗姆重复 (CRISPR) /与CRISPR相关的蛋白质 (Cas) 基因编辑显示出治疗危及视力的疾病的前景. 目前正在进行的临床试验和未来的研究旨在克服安全有效的眼疗法所面临的挑战.

关键词:
这就是CRISPR/CasPR.基因输送 基因输送 基因输送基因治疗是一种基因疗法.基因组编辑 基因组编辑眼部疾病 眼部疾病

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科学领域:

  • 眼科医生 眼科 眼科
  • 遗传学 是一个遗传学.
  • 生物技术是生物技术.

背景情况:

  • 危及视力的疾病需要新的治疗策略.
  • 克里斯普尔/卡斯基因编辑为治疗眼部疾病提供了潜在的潜力.
  • 克里斯帕/卡斯 (CRISPR/Cas) 促进了疾病建模和病原性研究.

研究的目的:

  • 审查CRISPR/Cas在眼部疾病模型和治疗中的应用.
  • 总结CRISPR/Cas的传递方法和策略.
  • 讨论目前的临床试验以及眼科中CRISPR/Cas的未来方向.

主要方法:

  • 对眼部疾病的CRISPR/Cas进行的临床前和临床研究的审查.
  • 基于CRISPR/Cas的疾病模型 (动物,有机体,细胞系) 的分析.
  • 检查治疗点,包括视网膜色素炎,玻璃眼和角膜疾病.

主要成果:

  • 克里斯普尔/卡斯已经被用来开发疾病模型和探索治疗应用.
  • 临床前研究显示了恢复细胞功能和减轻疾病表型的潜力.
  • 活跃的临床试验针对的是诸如玻璃眼,遗传性视网膜退化和与年龄相关的黄斑退化等疾病.

结论:

  • 克里斯普尔/卡斯技术在治疗一系列眼部疾病方面具有重大前景.
  • 需要解决诸如非目标效应,免疫性和监管障碍等挑战.
  • 未来的研究应该专注于提高特异性,疗效和成功临床转换的交付.