Omenn syndrome
Omenn syndrome is a severe, inherited immunodeficiency disorder characterized by a lack of immune protection and an autoimmune reaction that attacks the body's own tissues.
Overview
Omenn syndrome is a form of severe combined immunodeficiency (SCID). Individuals with this condition have virtually no immune protection from bacteria, viruses, and fungi, making them prone to repeated, life-threatening infections. Infants typically experience pneumonia and chronic diarrhea.
In addition to immunodeficiency, children develop autoimmunity, where the immune system attacks the body's own tissues. Features include:
- Very red skin (erythroderma)
- Hair loss (alopecia)
- Enlarged liver and spleen (hepatosplenomegaly)
- Enlargement of the thymus and lymph nodes
Without treatment to restore immune function, children with Omenn syndrome usually survive only until age 1 or 2.
Causes
Omenn syndrome is caused by mutations in several genes involved in immune function, most frequently the RAG1 and RAG2 genes. These genes provide instructions for proteins active in B cells and T cells (types of lymphocytes). These proteins help increase the diversity of surface proteins on lymphocytes, which allows the immune system to recognize a wide variety of foreign invaders.
Mutations in RAG1 and RAG2 drastically reduce protein function, severely limiting the diversity of B and T cells. While the number of T cells may be normal, they are highly similar and abnormal, leading them to attack the body's own cells and tissues, which accounts for the autoimmune features of the syndrome.
Inheritance
This condition is inherited in an autosomal recessive pattern, meaning both copies of the gene in each cell have mutations. The parents of an affected individual each carry one copy of the mutated gene but typically do not show signs or symptoms of the condition.
