在血液形成干细胞中进行ex vivo基因和细胞疗法
Irina O Petrova1, Svetlana A Smirnikhina1
1Laboratory of Genome Editing, Research Centre for Medical Genetics, Moskvorechye 1, 115478 Moscow, Russia.
International journal of molecular sciences
|December 11, 2025
概括
使用造血干细胞/原始细胞 (HSPCs) 的ex vivo基因疗法对遗传性疾病显示出有前途. 虽然lentiviral基因疗法是有效的,但由于瘤性风险,仔细的安全性分析至关重要.
科学领域:
- 生物技术是生物技术.
- 遗传学 是一个遗传学.
- 细胞疗法细胞疗法
背景情况:
- 活体细胞和基因疗法为遗传疾病提供了一个有前途的治疗策略.
- 造血干细胞/原始细胞 (HSPC) 是工程细胞疗法的主要焦点.
- 已批准的HSPC治疗方法是当前医疗应用的核心.
研究的目的:
- 审查已批准的ex vivo HSPC基因疗法方法.
- 分析治疗成功和潜在缺点的关键因素.
- 为了平衡基因疗法开发中的安全性和效率.
主要方法:
- 专注于将lentiviral基因整合到目标细胞基因组中.
- 对各种疾病批准的ex vivo基因疗法病例的分析.
- 评估安全问题,包括致癌潜力和载体设计.
主要成果:
- 隐形病毒载体是非常有效的,特别是对于不分裂的细胞.
- 病毒基因整合显示出显著的治疗效果.
- 自主无活化载体提高了安全性,但瘤发生风险仍然存在.
结论:
- 活体HSPC基因治疗是一种可行的遗传疾病治疗方法.
- 将治疗效率与安全平衡至关重要.
- 需要对整合场所进行持续严格的分析,以减轻致癌风险.
相关概念视频
Stem Cell Therapy for Tissue Regeneration
4.6K
Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
4.6K
Gene Therapy
27.3K
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...
27.3K
Regulation of Hematopoietic Stem Cells
3.9K
All blood and immune cells are produced from the multipotent hematopoietic stem cells (HSCs) by the process of hematopoiesis. However, they all have a limited life span. In addition, many are depleted in immune surveillance or combatting an injury or infection. This makes blood one of the most regenerative tissues. Hematopoiesis helps replenish these blood and immune cells, restoring the body's normal functioning. However, overproduction of blood and immune cells can make them cancerous or...
3.9K
Bone Marrow Sampling and Transplants
816
Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
816
Multipotency of Hematopoietic Stem Cells
3.8K
The hematopoietic stem cells or HSCs are multipotent, meaning they can differentiate and give rise to all blood and immune cells. HSCs are maintained in the quiescent stage until an external stimulus initiates their differentiation. The multipotent HSCs exist as two heterogeneous populations, long-term repopulating cells (LTRC) and short-term repopulating cells (STRC). The two HSC populations have different surface markers or receptors and are classified based on quiescence and long-term...
3.8K
Stem Cell Culture
6.0K
Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
6.0K


