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相关概念视频

Gene Therapy00:59

Gene Therapy

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
Cancer-Critical Genes II: Tumor Suppressor Genes01:05

Cancer-Critical Genes II: Tumor Suppressor Genes

Genes usually encode proteins necessary for the proper functioning of a healthy cell. Mutations can often cause changes to the gene expression pattern, thereby altering the phenotype.
When the function of certain critical genes, especially those involved in cell cycle regulation and cell growth signaling cascades, gets disrupted, it upsets the cell cycle progression. Such cells with unchecked cell cycles start proliferating uncontrollably and eventually develop into tumors.
Such genes that act...
Tumor Immunotherapy01:27

Tumor Immunotherapy

Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.

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相关实验视频

Updated: Jul 18, 2026

Tumor Engraftment in a Xenograft Mouse Model of Human Mantle Cell Lymphoma
10:52

Tumor Engraftment in a Xenograft Mouse Model of Human Mantle Cell Lymphoma

Published on: March 30, 2018

基因疗法:治疗性基因导致淋巴瘤.

Niels-Bjarne Woods1, Virginie Bottero, Manfred Schmidt

  • 1Laboratory of Genetics, The Salk Institute for Biological Studies, La Jolla, California 92037, USA.

Nature
|April 28, 2006
PubMed
概括

使用IL2RG基因治疗X关联严重联合免疫缺陷 (X-SCID) 的基因治疗可能会带来风险. 研究表明,治疗基因本身可以在小鼠模型中促进T细胞淋巴瘤的发展.

科学领域:

  • 免疫学 免疫学 免疫学
  • 基因治疗 基因治疗
  • 在瘤学瘤学.

背景情况:

  • 使用造血干细胞的基因疗法已经显示出治疗X-SCID (X-SCID) 的承诺.
  • 然而,最近的试验已经在治疗患者中发现T细胞白血病的发展,这促使重新评估这种方法的安全性.

研究的目的:

  • 研究IL2RG基因的潜在致癌作用,IL2RG基因是X-SCID基因治疗中使用的校正治疗基因.
  • 在临床前模型中评估与IL2RG基因治疗相关的T细胞淋巴瘤发展风险.

主要方法:

  • 一个小鼠模型被用来模拟X-SCID的基因治疗.
  • 该研究的重点是IL2RG基因作为治疗剂的影响.

主要成果:

  • 发现IL2RG基因有助于T细胞淋巴瘤的发展,在大量 (三分之一) 的受治疗动物中.
  • 这一发现挑战了之前的假设,即IL2RG具有最小的致癌性.

结论:

  • 使用IL2RG基因进行X-SCID的基因治疗可能会带来诱导T细胞淋巴瘤的固有风险.
  • 这些发现需要仔细重新评估X-SCID基因疗法试验的安全协议和风险效益分析.

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Tumor Engraftment in a Xenograft Mouse Model of Human Mantle Cell Lymphoma
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Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
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A Nonviral Approach to Generate Transient Chimeric Antigen Receptor T Cells Using mRNA for Cancer Immunotherapy

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