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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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相关实验视频

Updated: Jun 26, 2025

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
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针对T细胞工程的向基因传递系统.

Fengling Wang1, Yong Huang1, JiaQian Li1

  • 1Department of Biotherapy, State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University, Chengdu, 610041, People's Republic of China.

Cellular oncology (Dordrecht, Netherlands)
|May 16, 2024
PubMed
概括

利用T淋巴细胞进行治疗需要先进的基因传递. 本综述探讨病毒和非病毒载体,重点关注体内T细胞修饰以改善免疫疗法.

关键词:
非病毒载体的非病毒载体.基于T细胞的免疫疗法.有针对性的基因传递系统.病毒载体病毒载体

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A GMP-Compliant Procedure for the Generation of Gene-Modified T cells
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Clinical Application of Sleeping Beauty and Artificial Antigen Presenting Cells to Genetically Modify T Cells from Peripheral and Umbilical Cord Blood
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科学领域:

  • 免疫学 免疫学 免疫学
  • 生物技术是生物技术.
  • 基因治疗 基因治疗

背景情况:

  • T淋巴细胞对于宿主防御病原体和瘤至关重要.
  • 基因工程T细胞,如CAR-T和TCR-T,在治疗癌症和自身免疫性疾病方面表现有前途.
  • 目前的T细胞工程依赖于复杂的体外隔离和修饰,延迟治疗的发展.

研究的目的:

  • 审查T细胞的基因传递系统,包括病毒和非病毒载体.
  • 评估体内T细胞基因修饰策略的潜力.
  • 为推进基于T细胞的免疫疗法提供见解.

主要方法:

  • 对T细胞的病毒基因传递载体的分析.
  • 对T细胞的非病毒基因传递载体的评估.
  • 对体内T细胞基因修饰系统的当前研究进行审查.

主要成果:

  • 病毒载体提供高转导效率,但面临免疫性和生产挑战.
  • 非病毒载体更安全,更可扩展,但通常具有较低的转导效率.
  • 针对性地在体内将基因转移到T细胞中,而无需隔离,是显著的进步.

结论:

  • 为T细胞开发高效的体内基因传递系统对于未来的免疫疗法至关重要.
  • 克服当前病毒和非病毒载体的局限性是必不可少的.
  • 在体内T细胞修饰具有治疗疾病的革命性潜力.