Mechanism-Based Precision Therapy for the Treatment of Primary Immunodeficiency and Primary Immunodysregulatory Diseases.

Advances in understanding the mechanism, immunobiology, and pathophysiology of primary immunodeficiency diseases have created opportunities for the use of precision medicines for the treatment of disease-related manifestations. Modulation of the immune system to treat autoimmunity began with the use of intravenous immunoglobulin, improved with the development of monoclonal antibodies, and has now become standard in certain diseases with mechanistic-based targets that alter the molecular mechanism of disease. In this article, we review targeted therapies for disorders of hyperinflammation, primary immunodysregulatory diseases, and primary immunodeficiencies. We also look to the future where gene editing will tailor therapy in an even more precise way for each individual disease and patient.