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Related Concept Videos

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.
There are three main causes of immunodeficiency...
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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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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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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.
There are several types of targeted therapies against...
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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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Updated: Nov 25, 2025

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
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Gene therapy for primary immunodeficiencies.

Thomas A Fox1,2,3, Claire Booth3,4

  • 1University College London (UCL) Institute of Immunity and Transplantation, UCL, London, UK.

British Journal of Haematology
|December 18, 2020
PubMed
Summary
This summary is machine-generated.

Hematopoietic stem cell gene therapy (HSC GT) offers a promising alternative to allogeneic transplantation for primary immunodeficiencies (PIDs). While challenges like insertional mutagenesis and off-target effects exist, HSC GT is advancing rapidly, with a licensed product now available for ADA-SCID.

Keywords:
gene therapyprimary immunodeficienciesstem cell transplantation

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Area of Science:

  • Immunology
  • Genetics
  • Hematology

Background:

  • Primary immunodeficiencies (PIDs) are rare inherited immune disorders requiring definitive treatment.
  • Allogeneic haematopoietic stem cell transplantation (alloHSCT) is curative but carries risks like graft failure and graft-versus-host disease (GvHD).
  • Autologous haematopoietic stem cell gene therapy (HSC GT) aims to correct genetic defects without allogeneic complications.

Purpose of the Study:

  • To review the development and current clinical status of HSC GT for PIDs.
  • To discuss the potential of new gene editing technologies.
  • To outline future directions in the field of HSC GT for PIDs.

Main Methods:

  • Review of existing literature on HSC GT for PIDs.
  • Analysis of clinical trial data and outcomes.
  • Discussion of emerging gene editing technologies and their application.

Main Results:

  • HSC GT has been in development for two decades, with a licensed product for ADA-SCID now available.
  • Gene editing technologies offer potential solutions to limitations of viral gene addition.
  • HSC GT shows promise but requires individualized approaches and carries risks such as insertional mutagenesis and off-target toxicities.

Conclusions:

  • HSC GT is a rapidly advancing and promising therapeutic strategy for PIDs.
  • Further research and development are needed to optimize safety and efficacy.
  • The field is evolving with new technologies offering improved therapeutic potential for rare genetic immune disorders.