Inherited Immune System Disorders Stem Cell Therapy Development
Inherited immune system disorders, also known as primary immunodeficiency disorders (PIDs), are a group of rare genetic conditions that affect the body's immune system. Unlike acquired immune deficiencies, which are caused by external factors such as infections or medical treatments, inherited immune system disorders are present from birth and result from defects in genes involved in the development or functioning of the immune system. Stem cell therapy has emerged as a promising treatment approach for certain inherited immune system disorders, therefore, CD BioSciences offers stem cell therapy development services for PIDs.
Introduction into Inherited Immune System Disorders
The immune system is responsible for defending the body against harmful pathogens such as bacteria, viruses, and fungi. It is composed of various cells, tissues, and organs that work together to recognize and eliminate foreign invaders while maintaining tolerance to self-tissues. In individuals with inherited immune system disorders, this defense mechanism is compromised, leading to increased susceptibility to infections, recurrent infections, and other associated health problems.
There are over 400 different types of primary immunodeficiency disorders identified so far, each with its own specific genetic cause and clinical manifestations. Some of the most common inherited immune system disorders include:
| Severe combined immunodeficiency (SCID) | This is a severe disorder where both the T cells and B cells of the immune system are affected. It is characterized by a near absence or complete lack of immune function, making affected individuals highly susceptible to severe and life-threatening infections. |
| Common variable immunodeficiency (CVID) | CVID is a group of disorders characterized by low levels of immunoglobulins (antibodies) in the blood, leading to recurrent infections, particularly in the respiratory and gastrointestinal tracts. It typically presents in adolescence or adulthood. |
| X-linked agammaglobulinemia (XLA) | This disorder mainly affects males and is caused by a mutation in the BTK gene, leading to the absence of mature B cells and a severe deficiency in immunoglobulins. It results in recurrent bacterial infections, particularly in the respiratory system. |
| Chronic granulomatous disease (CGD) | CGD is characterized by defects in the ability of immune cells called phagocytes to kill certain types of bacteria and fungi. This leads to recurrent and severe infections, as well as the formation of granulomas (masses of immune cells) in various organs. |
| Wiskott-Aldrich syndrome (WAS) | WAS primarily affects males and is characterized by abnormalities in platelets, T cells, and B cells. It leads to recurrent infections, abnormal bleeding, eczema, and an increased risk of certain cancers. |
Stem Cell Therapy for Inherited Immune System Disorders
Stem cell therapy has emerged as a promising treatment approach for certain inherited immune system disorders. The goal of stem cell therapy is to replace or repair the defective immune cells or components in the patient's body, restoring their immune function and reducing susceptibility to infections.
Bone marrow transplantation or gene therapy can be useful to correct inherited immune system disorders. The success of stem cell therapy depends on several factors, including the specific disorder being treated, the patient's overall health, the source and quality of stem cells, the matching of donor and recipient (in allogeneic HSCT), and post-transplant care. Close monitoring of the patient's immune function and potential complications, as well as long-term follow-up, are crucial for ensuring the effectiveness and safety of stem cell therapy. Bone marrow donor and recipient must share at least some major histocompatibility complex (MHC) molecules to restore immune function. In an allogeneic bone marrow transplant, the donor marrow cells share some MHC molecules with the recipient.
Our Services
CD BioSciences offers stem cell therapy development services for PIDs, based on our advanced technology and professional team. Our services are provided in inherited immune system disorders s included but not limited as follows:
- Severe Combined Immunodeficiency (SCID)
- Common Variable Immunodeficiency (CVID)
- X-Linked Agammaglobulinemia (XLA)
- Chronic Granulomatous Disease (CGD)
- Wiskott-Aldrich Syndrome (WAS)
- Hyper IgM Syndrome
As a pioneer in biotechnology, CD BioSciences has grown into one of the largest independent biotechnology companies in the world. CD BioSciences is committed to providing professional and efficient service to our customers around the world. If you are interested in our service, please contact us.
Reference
- Janeway CA Jr, Travers P, Walport M, et al. Immunobiology: The Immune System in Health and Disease. 5th edition. New York: Garland Science; 2001.
For research use only, not for clinical use.
