Precision Therapy for the Treatment of Primary Immunodysregulatory Diseases

Abstract

Precision therapy is a concept in which medical treatment is tailored to the patient's individual needs based on individual characteristics and mechanism of disease. Primary immunodysregulatory disorders are an expanding group of primary immunodeficiency diseases that are characterized by early onset autoimmunity and autoinflammation. Precision therapy allows for the alteration of the aberrant immune response leading to clinical improvement of disease related manifestations. This article reviews targeted precision-based therapy for treatment of cytotoxic T-lymphocyte antigen haploinsufficiency, lipopolysaccharide-responsive beige-like anchor deficiency, activated PI3K deficiency syndrome, signal transducer and activator of transcription- 1 and -3 - gain-of-function disorders, and disorders of inflammasome activation.

Keywords: Biologic modifiers; Cytotoxic T-lymphocyte antigen 4; Hemophagocytic lymphohistiocytosis; Lipopolysaccharide-responsive beige-like anchor; Precision therapy; Signal transducer and activator of transcription 1 gain of function; Signal transducer and activator of transcription 3 gain of function.