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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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Gene Therapy00:59

Gene Therapy

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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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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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Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

1.3K
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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Related Experiment Video

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

Fabien Touzot1, Salima Hacein-Bey-Abina, Alain Fischer

  • 1U768 INSERM , Paris , France.

Expert Opinion on Biological Therapy
|May 15, 2014
PubMed
Summary

Gene therapy offers a promising treatment for primary immunodeficiencies (PIDs) by correcting T cell defects. Newer, safer vectors aim to improve efficacy and reduce genotoxicity risks associated with earlier gene transfer methods.

Keywords:
Wiskott–Aldrich syndromeadenosine deaminasechronic granulomatous diseasegene therapylentiviral vectorsprimary immunodeficiencysevere combined immunodeficiency

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Area of Science:

  • Immunology
  • Genetics
  • Molecular Biology

Background:

  • Gene therapy has become a viable treatment for primary immunodeficiencies (PIDs).
  • Ex vivo gene transfer into hematopoietic stem cells (HSCs) has corrected T cell immunodeficiency in severe combined immunodeficiency (SCID) forms.
  • Early retroviral vectors caused genotoxicity, prompting development of safer self-inactivating vectors.

Purpose of the Study:

  • To review updated gene therapy trial results for PIDs.
  • To detail advances in gene transfer and repair technologies.
  • To discuss future directions for PID management.

Main Methods:

  • Review of early studies and ongoing clinical trials for PID gene therapy.
  • Analysis of gene transfer and repair technologies.
  • Discussion of clinical experiences and future strategies.

Main Results:

  • Gene therapy has shown success in treating specific PIDs like SCID-X1 and adenosine deaminase deficiency.
  • Genotoxicity from first-generation retroviral vectors has been a concern.
  • Safer lentiviral vectors are being developed for improved safety and efficacy.

Conclusions:

  • Optimized gene therapy, particularly lentiviral transduction, shows potential as an alternative to allogeneic HSC transplantation for PIDs.
  • Gene therapy may become a leading treatment for PIDs in the near future.
  • Continued research focuses on enhancing safety and efficacy for broader PID management.