Primary immunodeficiencies (PID) are disorders that for the most part result from mutations
in genes involved in immune host defense and immunoregulation. These conditions are …

Monogenic diseases of the immune system, also known as inborn errors of immunity, are
caused by single-gene mutations resulting in immune deficiency and dysregulation. More …

Gene transfer into autologous hematopoietic stem progenitor cells (HSPCs) has the
potential to cure monogenic inherited disorders caused by an altered development and/or …

Chimeric antigen receptor T-cell (CAR-T) therapy has been approved for relapsed/refractory
B-cell lymphomas and greatly improves disease outcomes. The impressive success has …

Background Biallelic variations in the dedicator of cytokinesis 8 (DOCK8) gene cause a
combined immunodeficiency with eczema, recurrent bacterial and viral infections, and …

Single-cell molecular tools have been developed at an incredible pace over the last five
years as sequencing costs continue to drop and numerous molecular assays have been …

Background Primary immunodeficiency disease (PID) is a disorder caused by an inherited
flaw in the immune system that increases the susceptibility to infections. Methods In this …

Introduction: The transcription factors signal transducer and activator of transcription (STAT)
1 and STAT3 fulfill fundamental functions in nonimmune and immune cells. The description …

Advances in understanding the mechanism, immunobiology, and pathophysiology of
primary immunodeficiency diseases have created opportunities for the use of precision …

Abstract Purpose of Review Hematopoietic cell transplantation (HCT) is an established
curative treatment for children with primary immunodeficiencies. This article reviews the …