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Related Concept Videos

Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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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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Immunodeficiency Diseases01:25

Immunodeficiency Diseases

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Immunodeficiency disorders are conditions in which the immune system's ability to fight infectious disease and cancer is compromised or entirely absent. The immune system comprises a complex network of cells, tissues, and organs that work together to protect the body from potentially harmful invaders. When this system is deficient or not functioning properly, it leaves the body susceptible to infections, diseases, or other complications.
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Tumor Immunotherapy01:27

Tumor Immunotherapy

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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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Targeted Cancer Therapies02:57

Targeted Cancer Therapies

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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.
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Development of Immunocompetence01:22

Development of Immunocompetence

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The initiation of cell-mediated immunity can be observed as early as the third month of fetal growth, with active antibody-mediated immunity following approximately one month later.
The initial cells that migrate from the fetal thymus settle within the skin and epithelial tissues lining the mouth, digestive tract, and in females, the uterus and vagina. These cells, including skin-based dendritic cells, serve as antigen-presenting cells, playing a key role in T cell activation.
Subsequent T...
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Related Experiment Video

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Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
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Gene therapy for primary immunodeficiencies.

Christine Rivat1, Giorgia Santilli, H Bobby Gaspar

  • 1UCL Institute of Child Health, Centre for Immunodeficiency, London WCIN 1EH, United Kingdom.

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Gene therapy offers a promising alternative to stem cell transplants for primary immunodeficiencies (PIDs). This approach shows clinical efficacy, especially for patients lacking HLA-matched donors, advancing PID treatment options.

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Area of Science:

  • Immunology
  • Hematology
  • Genetics

Background:

  • Primary immunodeficiencies (PIDs) have historically relied on allogeneic hematopoietic stem cell transplantation (HSCT).
  • Recent advancements include ex vivo somatic gene therapy using autologous cells.

Purpose of the Study:

  • To review the current status of gene therapy trials for PIDs.
  • To discuss the application of gene therapy strategies to a broader range of PIDs.

Main Methods:

  • Review of existing clinical trial data for gene therapy in PIDs.
  • Analysis of preclinical models and technological advancements.

Main Results:

  • Ex vivo somatic gene therapy demonstrates significant clinical efficacy.
  • Gene therapy is particularly beneficial for patients lacking HLA-matched stem cell donors or at high risk of allogeneic transplant toxicity.

Conclusions:

  • Gene therapy represents a safer and more sophisticated approach for treating PIDs.
  • The application of gene therapy strategies is expanding to encompass a wider spectrum of PIDs.