Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Gene Therapy00:59

Gene Therapy

26.8K
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...
26.8K
Immunodeficiency Diseases01:25

Immunodeficiency Diseases

1.5K
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...
1.5K
Tumor Immunotherapy01:27

Tumor Immunotherapy

876
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.
876
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

8.1K
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...
8.1K
Development of Immunocompetence01:22

Development of Immunocompetence

586
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...
586
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

676
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.
676

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Ethical and Clinical Boundaries in Genomics & Newborn Screening: A Brief Report from IPIC2025.

International journal of neonatal screening·2026
Same author

Topical JAK inhibitor for refractory skin inflammation in STAT1 GOF.

Journal of human immunity·2026
Same author

Precision medicine in paediatrics: Progress and priorities.

British journal of clinical pharmacology·2026
Same author

Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.

Molecular therapy : the journal of the American Society of Gene Therapy·2026
Same author

International clinical consensus on leukocyte adhesion deficiency-I: Modified Delphi analysis.

The journal of allergy and clinical immunology. Global·2026
Same author

Functional Characterization of an IL2RG Variant, a Case Report of X-Linked T- B + NK + SCID.

Immunity, inflammation and disease·2025

Related Experiment Video

Updated: Nov 22, 2025

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

17.5K

Gene Therapy for Primary Immunodeficiency.

Benjamin C Houghton1, Claire Booth1,2

  • 1Molecular and Cellular Immunology, UCL Great Ormond Street Institute of Child Health, London, United Kingdom.

Hemasphere
|January 6, 2021
PubMed
Summary

Gene therapy using lentiviral vectors has advanced significantly, offering effective treatments for immune deficiencies. Current approaches show enhanced safety and efficacy, with new gene editing technologies emerging for complex genetic disorders.

More Related Videos

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
11:16

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

7.9K
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

11.4K

Related Experiment Videos

Last Updated: Nov 22, 2025

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

17.5K
Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
11:16

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

7.9K
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

11.4K

Area of Science:

  • * Immunology and Genetics
  • * Biotechnology and Therapeutics

Background:

  • * Significant advancements in gene therapy over 30 years.
  • * Lentiviral vector transduction of hematopoietic stem cells enables effective immune reconstitution.
  • * Historical challenges included adverse events impacting clinical efficacy.

Purpose of the Study:

  • * To review key developments in gene therapy for primary immune deficiency.
  • * To highlight the transition from experimental to commercialized therapeutic products.
  • * To discuss emerging gene editing technologies and their potential.

Main Methods:

  • * Review of historical and recent scientific literature on gene therapy for primary immune deficiency.
  • * Analysis of technological progress in lentiviral vectors and ex vivo hematopoietic stem cell transduction.
  • * Examination of preclinical studies and gene editing technologies.

Main Results:

  • * Gene therapy has evolved into a safe and effective treatment for primary immune deficiency.
  • * Commercialized products demonstrate enhanced safety and efficacy compared to earlier experimental therapies.
  • * Preclinical studies show promise for challenging targets, and gene editing offers new therapeutic avenues.

Conclusions:

  • * Gene therapy, particularly using lentiviral vectors, is a viable and improving treatment for primary immune deficiencies.
  • * Ongoing research and emerging technologies like gene editing are expanding therapeutic possibilities.
  • * Focus is shifting towards addressing complex genetic disorders and improving gene regulation for efficacy.