Related Experiment Video
Updated: Dec 5, 2025
08:52
Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
Published on: May 27, 2011
17.6K
Gene therapy for primary immunodeficiencies: up-to-date.
Kritika Chetty1,2, Claire Booth1,2
1Department of Infection, Immunity and Inflammation, UCL Great Ormond Street Institute of Child Health, London, United Kingdom.
Expert Opinion on Biological Therapy
|October 20, 2020
Summary
Gene therapy offers a promising curative treatment for primary immunodeficiencies (PIDs), overcoming limitations of traditional stem cell transplants. Advances in vector design and gene editing enhance safety and efficacy for PIDs.
Area of Science:
- Immunology
- Genetics
- Biotechnology
Background:
- Primary immunodeficiencies (PIDs) are genetic disorders increasing infection susceptibility.
- Hematopoietic stem cell transplant (HSCT) is curative but carries significant risks.
- Gene therapy using autologous stem cells presents a safer, targeted alternative.
Purpose of the Study:
- To review advancements in gene therapy and gene editing for PIDs.
- To summarize current gene therapy applications in specific PIDs.
Main Methods:
- Review of scientific literature on gene therapy and gene editing.
- Analysis of historical and recent clinical trial data.
- Examination of vector design improvements and cryopreservation techniques.
Main Results:
- Gene therapy has evolved significantly, with licensed products now available.
- Improved vector design has mitigated risks like insertional oncogenesis.
- Cryopreservation enhances gene therapy accessibility.
- Gene editing technologies show future potential for PID treatment.
Conclusions:
- Gene therapy is becoming a standard of care for select PIDs.
- Ongoing advancements in gene therapy and editing promise broader applications.
- These innovations offer improved outcomes for patients with PIDs.
More Related Videos
Related Concept Videos
00:59Gene Therapy
27.0K
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...
27.0K
01:25Immunodeficiency Diseases
1.8K
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...
There are three main causes of immunodeficiency...
1.8K
01:27Tumor Immunotherapy
1.3K
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.
1.3K
01:29Microorganisms in Medicine and Therapeutics
744
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.
744
02:57Targeted Cancer Therapies
8.3K
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...
There are several types of targeted therapies against...
8.3K
01:22Development of Immunocompetence
606
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...
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...
606