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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.
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.
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...

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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 primary immunodeficiencies.

Alain Fischer1, S Hacein-Bey-Abina, M Cavazzana-Calvo

  • 1Developpement Normal et Pathologique du Systeme Immunitaire, INSERM U 768, Hopital Necker, 149 rue de sevres, Paris 75015, France. alain.fischer@inserm.fr

Immunology and Allergy Clinics of North America
|May 25, 2010
PubMed
Summary
This summary is machine-generated.

Gene therapy offers a promising approach to correct inherited diseases by replacing mutated genes. Its feasibility is now well-established in treating primary immunodeficiencies (PIDs), supporting its use for these conditions.

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Area of Science:

  • Genetics
  • Immunology
  • Medical Science

Background:

  • Gene therapy aims to correct monogenic inherited diseases by introducing functional gene copies.
  • Implementing gene therapy has presented significant challenges.
  • Primary immunodeficiencies (PIDs) represent a key area where gene therapy has demonstrated feasibility.

Purpose of the Study:

  • To review the concept and application of gene therapy in treating genetic disorders.
  • To highlight the successful application of gene therapy in primary immunodeficiencies.
  • To present the rationale for using gene therapy in managing certain PIDs.

Main Methods:

  • Conceptual review of gene therapy principles.
  • Analysis of clinical outcomes in PID patients treated with gene therapy.
  • Synthesis of evidence supporting gene therapy for PIDs.

Main Results:

  • Gene therapy has proven feasible for correcting genetic defects.
  • Significant progress has been made in applying gene therapy to PIDs.
  • Successful correction of genetic mutations has been observed in PID patients.

Conclusions:

  • Gene therapy is a viable strategy for treating monogenic inherited diseases.
  • The success in PIDs provides a strong foundation for further gene therapy applications.
  • Further research and application of gene therapy for PIDs are warranted.