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Gene Therapy

Significant progress achieved over the past decade has turned gene therapy into a tangible and effective approach for treating and curing primary immunodeficiencies (PIDs).

What is Gene Therapy?

Gene therapy, akin to a ‘mini gene transplant,’ involves extracting stem cells from patients with primary immunodeficiency (PID), replacing the defective genes within these cells with healthy and fully functional genes, and reintroducing the gene-corrected cells back into the patient’s body. This process enables the cells to generate the necessary cells or proteins essential for …

Thymus Transplantation for congenital absence of the thymus (athymia)

The Significance of the Thymus Gland

Situated in the upper chest, just in front of the heart, the thymus gland is a vital organ. Its importance lies in facilitating the normal development of specific white blood cells known as T-cells. Stem cells, originating in the bone marrow, migrate to the thymus, where they undergo maturation into functional T cells capable of combating infections. T-cells are pivotal components of the immune system, making the thymus instrumental in instructing the immune system to safeguard the body against the everyday germs in our environment.

Bone Marrow Transplantation

Bone marrow transplantation (BMT) stands as a cure for numerous primary immunodeficiency (PID) conditions. Over the past two decades, significant strides have been made in enhancing success and survival rates, expanding the spectrum of treatable conditions through BMT.

What is Bone Marrow?

Bone marrow, located at the core of bones, is the soft, spongy tissue crucial for generating three vital blood cell types: red blood cells, white blood cells (integral to the immune system), and platelets. Stem cells, the initial form of these cells, undergo maturation within the bone marrow before being …

Antibiotics and Immunodeficiency

Antibiotics play a crucial role in managing and treating bacterial infections. This information is intended for individuals with primary immunodeficiency (PID) or secondary immunodeficiency (SID) and their caregivers. It aims to advocate for the safe and effective utilization of antibiotics and addresses some commonly asked questions.

Understanding Antibiotics

Antibiotics are medications designed to combat infections instigated by bacteria, a particular category of microorganisms responsible for illnesses. Their mechanism involves either eradicating the bacteria or impeding their growth. …