Advances in the treatment of severe combined immunodeficiency

Severe Combined Immunodeficiency (SCID) is the most profound inborn error of immunity affecting cellular and humoral immunity. Hematopoietic stem cell transplantation has been a curative treatment since 1968. Huge progress has been made since then in understanding the underlying genetics, improving outcomes from trans-plant, and introducing gene therapy in particular for adenosine deaminase deficient-and IL2 receptor gamma -deficient SCID. Newborn screening has been widely introduced across the world to enable definitive treatment before infection occurs. This article aims to review the latest evidence on how to achieve curative treatment with minimal short-and long-term toxicity, normal immune reconstitution and good quality of life.

Automated summary

This article reviews the latest evidence on achieving curative treatment with minimal short- and long-term toxicity, normal immune reconstitution, and good quality of life.