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Related Concept Videos

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...
Immunodeficiency Diseases01:25

Immunodeficiency Diseases

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.
There are three main causes of immunodeficiency disorders...
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.
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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.
Development of Immunocompetence01:22

Development of Immunocompetence

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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Updated: Jul 5, 2026

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
08:52

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant

Published on: May 27, 2011

Gene therapy for primary immunodeficiencies.

Adrian J Thrasher1

  • 1Centre for Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, University College London and Great Ormond Street Hospital for Children NHS Trust, 30 Guilford Street, London WC1N 1EH, UK. a.thrasher@ich.ucl.ac.uk

Immunology and Allergy Clinics of North America
|April 22, 2008
PubMed
Summary
This summary is machine-generated.

Gene therapy offers a promising treatment for primary immunodeficiencies, addressing limitations of traditional stem cell transplants. Advances in vector technology are enhancing efficacy and safety for these genetic disorders.

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Area of Science:

  • Immunology
  • Genetics
  • Molecular Biology

Background:

  • Primary immunodeficiencies (PIDs) are genetic disorders impacting the immune system.
  • Conventional treatments like allogeneic stem cell transplantation have significant risks, especially in mismatched cases.
  • Gene therapy presents a potential alternative due to defined disease mechanisms and accessible hematopoietic stem cells.

Purpose of the Study:

  • To review the potential of gene therapy for treating primary immunodeficiencies.
  • To highlight the advantages of gene therapy over conventional transplantation.
  • To discuss recent advancements in gene therapy vectors and their implications.

Main Methods:

  • Review of clinical studies and technological developments in gene transfer for PIDs.
  • Analysis of gamma retroviral gene transfer efficacy and toxicity.
  • Evaluation of newer vector designs for improved safety and effectiveness.

Main Results:

  • Gamma retroviral gene transfer has demonstrated significant therapeutic benefits in clinical studies.
  • Gene therapy shows potential to overcome limitations associated with stem cell transplantation.
  • Emerging vector technologies offer solutions to mitigate treatment-related toxicities.

Conclusions:

  • Gene therapy is a highly promising and increasingly efficacious strategy for managing primary immunodeficiencies.
  • Ongoing advancements in vector design are crucial for optimizing gene therapy outcomes.
  • Gene therapy is poised to become a standard treatment for many PIDs.