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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...
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...
Cytotoxic T Cells-mediated Immune Response01:27

Cytotoxic T Cells-mediated Immune Response

Cytotoxic T cells are a vital component of the immune system. They have the remarkable ability to identify and target antigens on infected or abnormal cells. These antigens often originate from intracellular pathogens such as viruses or abnormal proteins cancer cells produce.
Immunological surveillance is the ability of immune cells to monitor and eliminate infected cells with intracellular pathogens, neoplastically transformed cells, and cells with non-self antigens. Cytotoxic T cells and NK...
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: Jun 12, 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 severe combined immunodeficiency].

Line Barrett Petersen1, Thomas G Jensen

  • 1Aarhus Universitet, Institut for Human Genetik, Denmark. line.barrett@studmed.au.dk

Ugeskrift for Laeger
|June 11, 2010
PubMed
Summary

Gene therapy for adenosine deaminase deficiency (ADA-SCID) shows promise, with most children no longer needing enzyme therapy and exhibiting improved immune function. Long-term safety is still being evaluated but initial results suggest a low risk of malignancy.

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Related Experiment Videos

Last Updated: Jun 12, 2026

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
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Transduction and Expansion of Primary T Cells in Nine Days with Maintenance of Central Memory Phenotype

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Area of Science:

  • * Hematology
  • * Immunology
  • * Gene Therapy

Context:

  • * Severe combined immunodeficiency (SCID) is a group of rare genetic disorders that affect the immune system.
  • * Adenosine deaminase (ADA) deficiency is a specific type of SCID that can be treated with enzyme replacement therapy or gene therapy.
  • * Gene therapy offers a potential alternative to lifelong enzyme replacement therapy for ADA-SCID.

Purpose:

  • * To review the outcomes of a clinical gene therapy trial for children with ADA-SCID.
  • * To evaluate the efficacy and safety of gene modified autologous hematopoietic stem cell transplantation.
  • * To assess the long-term impact on immune function and toxic metabolite levels.

Summary:

  • * Ten children with ADA-SCID were treated with gene-modified autologous hematopoietic stem cells.
  • * Eight patients discontinued enzyme-replacement therapy post-treatment.
  • * Nine patients demonstrated improved immune function and sustained low concentrations of toxic metabolites, with no observed clonal outgrowths, suggesting a limited risk of future malignancy.

Impact:

  • * Gene therapy provides a potentially curative treatment for ADA-SCID, reducing the need for continuous enzyme replacement.
  • * The treatment demonstrates significant improvements in immune reconstitution and metabolic control.
  • * While promising, ongoing monitoring is necessary to further improve the safety profile of gene therapy for SCID.