Inborn errors of immunity (IEI) are a group of rare heterogeneous diseases. Currently, more
than 400 monogenetic IEI have been identified and increasingly a genetic diagnosis can be …

This is the seventh special EBMT report on the indications for haematopoietic stem cell
transplantation for haematological diseases, solid tumours and immune disorders. Our aim …

Recent clinical trials of gene therapy have shown remarkable therapeutic benefits and an
excellent safety record. They provide evidence for the long-sought promise of gene therapy …

Background The Primary Immune Deficiency Treatment Consortium was formed to analyze
the results of hematopoietic-cell transplantation in children with severe combined …

Importance Newborn screening for severe combined immunodeficiency (SCID) using
assays to detect T-cell receptor excision circles (TRECs) began in Wisconsin in 2008, and …

This is the sixth special report that the European Society for Blood and Marrow
Transplantation regularly publishes on the current practice and indications for …

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 …

Importance Wiskott-Aldrich syndrome is a rare primary immunodeficiency associated with
severe microthrombocytopenia. Partially HLA antigen–matched allogeneic hematopoietic …

Innate lymphoid cells (ILCs) have potent immunological functions in experimental conditions
in mice, but their contributions to immunity in natural conditions in humans have remained …

Background In previous clinical trials involving children with X-linked severe combined
immunodeficiency (SCID-X1), a Moloney murine leukemia virus–based γ-retrovirus vector …