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相关概念视频

Gene Therapy00:59

Gene Therapy

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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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In-vitro Mutagenesis01:16

In-vitro Mutagenesis

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To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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CRISPR01:59

CRISPR

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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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相关实验视频

Updated: Jul 23, 2025

A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model
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胎儿基因治疗 胎儿基因治疗

Simon N Waddington1,2, William H Peranteau3, Ahad A Rahim4

  • 1EGA Institute for Women's Health, University College London, London, UK.

Journal of inherited metabolic disease
|July 20, 2023
PubMed
概括
此摘要是机器生成的。

胎儿基因疗法,曾经是一个遥远的概念,正在接近临床现实. 在产前诊断和胎儿手术方面的进展为在子宫内基因纠正遗传疾病铺平了道路.

关键词:
基因治疗的基因疗法遗传代谢性疾病遗传性代谢性疾病

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Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model
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Last Updated: Jul 23, 2025

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科学领域:

  • 医学科学 医学科学 医学科学
  • 遗传学 遗传学 是一个
  • 生物技术是生物技术.

背景情况:

  • 基因疗法已经从一个不成熟的概念演变为临床现实.
  • 治疗遗传疾病的趋势正在转向早期干预.
  • 遗传疾病的产前诊断正在迅速改善.

研究的目的:

  • 审查胎儿基因疗法的历史发展.
  • 讨论胎儿手术和诊断能力的进展.
  • 探索遗传代谢疾病的临床前基因疗法.

主要方法:

  • 基因治疗的历史审查.
  • 讨论胎儿手术和产前诊断方面的进展.
  • 对遗传代谢疾病的临床前数据的探索.

主要成果:

  • 自20世纪90年代末以来,基因疗法已经显著成熟.
  • 改善的产前诊断和胎儿手术使得更早的干预成为可能.
  • 临床前研究表明,胎儿基因疗法在代谢障碍中具有前景.

结论:

  • 胎儿基因疗法是一个正在发展中的领域,它有着明确的临床应用轨迹.
  • 技术和临床意志的融合支持了在子宫内基因治疗的进步.
  • 对临床前模型的进一步研究对于胎儿基因疗法的成功至关重要.