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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

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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 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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Related Experiment Video

Updated: Apr 17, 2026

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.

A Fischer1,2,3,4, S Hacein-Bey Abina5,6, F Touzot1,3,7,8

  • 1Paris Descartes - Sorbonne Paris Cité University, Imagine Institute, Paris, France.

Clinical Genetics
|February 25, 2015
PubMed
Summary

Gene therapy successfully treats primary immunodeficiencies (PID) by correcting genetic defects in hematopoietic stem cells. This approach offers a safe and effective treatment for severe combined immunodeficiencies (SCID) and other life-threatening conditions.

Keywords:
Wiskott-Aldrich syndromegene therapyprimary immunodeficienciesself-inactivating retrovirussevere combined immunodeficiency

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Area of Science:

  • Medicine
  • Genetics
  • Immunology

Background:

  • Gene therapy has emerged as a viable medical treatment, particularly for primary immunodeficiencies (PID).
  • Hematopoietic stem cells are accessible for genetic modification to correct immune cell differentiation defects.
  • Ex vivo gene transfer into hematopoietic cells can potentially correct disease phenotypes.

Purpose of the Study:

  • To review the application and safety of gene therapy for primary immunodeficiencies.
  • To highlight the development and efficacy of gene therapy in treating severe combined immunodeficiencies (SCID).
  • To discuss advancements in gene therapy vectors for improved safety and efficacy.

Main Methods:

  • Utilized retroviral vectors for ex vivo gene transfer into hematopoietic stem cells.
  • Developed enhancer-element-devoid retroviral vectors to mitigate genotoxicity risks.
  • Analyzed clinical trial data for safety and efficacy in PID treatment.

Main Results:

  • Sustained correction of T cell immunodeficiency in SCID patients for over a decade.
  • Demonstrated safety and efficacy of modified retroviral vectors in recent PID trials.
  • Observed genotoxicity issues with early retroviral vectors due to oncogene transactivation.

Conclusions:

  • Gene therapy is an established and effective treatment for certain primary immunodeficiencies.
  • Advanced retroviral vectors offer a safer and efficacious approach for PID gene therapy.
  • Future applications are anticipated for a wider range of life-threatening PID treatments.