Advanced therapy medicinal products (ATMPs) have a massive potential to address existing
unmet medical needs. Specifically, gene therapy medicinal products (GTMPs) may …

Autoimmune neutropenia of infancy (AIN), also called primary autoimmune neutropenia, is a
disease in which antibodies recognize membrane antigens of neutrophils, mostly located on …

Purpose Coronin-1A deficiency is a recently recognized autosomal recessive primary
immunodeficiency caused by mutations in CORO1A (OMIM 605000) that results in T-cell …

The evolutionary emergence of vertebrates was accompanied by the invention of adaptive
immunity. This is characterized by extraordinarily diverse repertoires of somatically …

Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the
blood and immune systems has placed them at the forefront of cell and gene therapy …

Background Absent T-cell immunity resulting in life-threatening infections provides a clear
rationale for hematopoetic stem cell transplantation (HSCT) in patients with severe …

Around 65% of primary immunodeficiencies are antibody deficiencies. Functional tests are
useful tools to study B‐cell functions in vitro. However, no accepted guidelines for …

Infectious complications are a major cause of morbidity and mortality in patients with primary
or secondary immunodeficiency. Prevention of infectious diseases by vaccines is among the …

Primary immunodeficiency diseases (PID) result from defects in genes affecting the immune
and other systems in many and varied ways (,). Until the last few years, treatments have …

IRAK-4 and MyD88 deficiencies impair interleukin 1 receptor and Toll-like receptor (TLR)
signaling and lead to heightened susceptibility to invasive bacterial infections. Individuals …