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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...
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.
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

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 specific...
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...

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Related Experiment Video

Updated: May 28, 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 primary immunodeficiency.

Claire Booth1, H Bobby Gaspar, Adrian J Thrasher

  • 1Molecular Immunology Unit, Centre of Immunodeficiency, Institute of Child Health, London, UK.

Current Opinion in Pediatrics
|October 6, 2011
PubMed
Summary

Gene therapy offers a promising alternative to haematopoietic stem cell transplantation (HSCT) for primary immunodeficiencies (PIDs) when a matched donor is unavailable. While demonstrating successful immune reconstitution, ongoing research addresses potential risks like vector insertion.

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Area of Science:

  • Immunology
  • Genetics
  • Pediatric Medicine

Background:

  • Haematopoietic stem cell transplantation (HSCT) is a primary treatment for primary immunodeficiencies (PIDs), but donor availability is a significant limitation.
  • Gene therapy using autologous gene-corrected stem cells has emerged as an alternative for patients lacking a suitable donor.
  • This approach has been utilized for over a decade in treating various PIDs, including severe combined immunodeficiency.

Purpose of the Study:

  • To review long-term outcome data from gene therapy clinical trials for PIDs.
  • To discuss significant adverse events associated with gene therapy for PIDs.
  • To explore current strategies for enhancing the efficacy and safety of PID gene therapy.

Main Methods:

  • Review of clinical trial data on gene therapy for primary immunodeficiencies.
  • Analysis of long-term outcomes and adverse events.
  • Discussion of ongoing research in viral vector development and gene editing.

Main Results:

  • Gene therapy has shown encouraging long-term outcomes for PIDs like chronic granulomatous disease and Wiskott-Aldrich syndrome.
  • Clinical trials confirm the feasibility of stem cell transduction and immune reconstitution.
  • Potential risks, including clonal disturbances from vector insertion, have been identified.

Conclusions:

  • Gene therapy is a viable alternative for PIDs when HSCT is not an option due to donor limitations.
  • Proof-of-principle for gene therapy in PIDs is established, with successful immune reconstitution observed.
  • Further research focuses on improving vector safety and efficacy, including regulated transgene expression and reduced mutagenic potential.