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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

Microorganisms in Medicine and Therapeutics

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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.
1.2K
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

9.0K
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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Development of Immunocompetence01:22

Development of Immunocompetence

1.0K
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: Feb 23, 2026

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
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Gene Therapy Approaches to Immunodeficiency.

Sujal Ghosh1, H Bobby Gaspar2

  • 1Infection, Immunity, Inflammation, Molecular and Cellular Immunology Section, University College London, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK; Department of Pediatric Oncology, Hematology and Clinical Immunology, Center of Child and Adolescent Health, Heinrich-Heine-University, Moorenstraße 5, 40225 Düsseldorf, Germany.

Hematology/Oncology Clinics of North America
|September 13, 2017
PubMed
Summary

Gene therapy using corrected stem cells offers a promising treatment for primary immunodeficiencies. Newer, safer viral vectors have improved outcomes and reduced risks in treating these rare genetic disorders.

Keywords:
Adenosine deaminase deficiencyChronic granulomatous diseaseGene therapyPrimary immunodeficiencyWiskott-Aldrich syndromeX-linked severe combined immunodeficiency

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Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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Area of Science:

  • * Hematology and Immunology
  • * Gene Therapy
  • * Genetic Disorders

Background:

  • * Gene-corrected autologous hematopoietic stem cell transplantation is a novel therapeutic strategy for primary immunodeficiencies.
  • * Early gene addition strategies using retroviral vectors showed promise but were complicated by malignancies.
  • * Safer viral vectors have been developed to mitigate genotoxicity risks.

Purpose of the Study:

  • * To review advancements in gene therapy for primary immunodeficiencies.
  • * To highlight progress in treating specific disorders like adenosine deaminase deficiency and X-linked severe combined immunodeficiency.

Main Methods:

  • * Review of clinical trials and therapeutic approaches over the last decade.
  • * Focus on gene-corrected autologous hematopoietic stem cell transplantation.
  • * Analysis of gene therapy strategies using improved viral vectors.

Main Results:

  • * Improved clinical outcomes in patients treated with newer gene therapy approaches.
  • * Successful application of gene therapy in adenosine deaminase deficiency, X-linked severe combined immunodeficiency, chronic granulomatous disease, and Wiskott-Aldrich syndrome.
  • * Reduced risk of genotoxicity and malignancies compared to earlier methods.

Conclusions:

  • * Gene therapy for primary immunodeficiencies has evolved significantly with safer vectors.
  • * Current gene therapy approaches demonstrate improved efficacy and safety.
  • * This therapeutic modality offers a viable alternative for patients lacking suitable donors.