Primary immunodeficiency disease (PID) is a group of inherited disorders that seriously affects children’s life and health

and causes a burden of families and the whole society. The main treatment strategy for PID in the past was replacement therapy or immune reconstruction by hematopoietic stem cell transplantation. In recent years

with the development of molecular biology

the pathogenesis of many cases of PID has been clarified. On this basis

precise and targeted therapy for defected molecules has been gradually carried out in clinical practice. The precise treatment of PID begins with the immunoglobulin G and enzyme replacement

and has been improved with the targeted small molecule inhibitors or biological macromolecules based on signal pathways. From the hematopoietic stem cell transplantation to gene therapy or gene editing in terms of cure strategy

this treatment strategy has been changing a lot

which indicates that PID as a typical indication

is bringing breakthroughs in precision medicine.