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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...
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...
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.

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Related Experiment Video

Updated: May 18, 2026

Transduction and Expansion of Primary T Cells in Nine Days with Maintenance of Central Memory Phenotype
08:49

Transduction and Expansion of Primary T Cells in Nine Days with Maintenance of Central Memory Phenotype

Published on: March 18, 2020

Gene therapy for primary immunodeficiencies: Part 1.

Marina Cavazzana-Calvo1, Alain Fischer, Salima Hacein-Bey-Abina

  • 1U768 INSERM, Paris, France. m.cavazzana@nck.aphp.fr

Current Opinion in Immunology
|September 18, 2012
PubMed
Summary

Hematopoietic stem cell gene therapy has benefited most patients with SCID-X1 or ADA-SCID. New trials focus on safer gene therapy vectors and editing technologies due to past safety concerns.

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Last Updated: May 18, 2026

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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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Published on: May 27, 2011

Area of Science:

  • Immunology
  • Genetics
  • Hematology

Background:

  • Severe Combined Immunodeficiency (SCID) encompasses genetic disorders impairing immune function.
  • Hematopoietic stem cell gene therapy has been explored for SCID-X1 and ADA-SCID.
  • Gammaretroviral vectors were used in early gene therapy trials.

Purpose of the Study:

  • To review gene therapy outcomes for SCID-X1 and ADA-SCID.
  • To discuss advancements in gene therapy safety and technology.
  • To compare gene therapy with allogeneic transplantation for SCID.

Main Methods:

  • Analysis of clinical trial data from over 60 patients treated with gene therapy.
  • Review of safety profiles and clinical benefits.
  • Exploration of new gene therapy vectors and gene editing technologies.

Main Results:

  • Gene therapy led to immune reconstitution and clinical benefit in most SCID-X1 and ADA-SCID patients.
  • Insertional oncogenesis was observed in SCID-X1 trials, prompting safety improvements.
  • Development of safer integrating vectors and gene editing technologies is ongoing.

Conclusions:

  • Gene therapy offers a viable treatment for SCID-X1 and ADA-SCID.
  • Ongoing research focuses on enhancing gene therapy safety and efficacy.
  • Gene therapy presents an alternative to allogeneic transplantation, with distinct advantages and disadvantages.