Ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPCs)
engineered with integrating vectors is a promising treatment for monogenic diseases, but …

Hematopoietic stem-cell gene therapy has proven to be an effective treatment for several
primary immunodeficiencies, and yet companies in this space are withdrawing from the EU …

Background Hematopoietic stem cell transplantation (HSCT) represents a curative treatment
for patients with severe combined immunodeficiency (SCID), a group of monogenic immune …

Newborn screening for severe combined immune deficiency (SCID) is the first inborn error of
immunity (IEI) to be detected through population screening. It also represents the first …

Adenosine deaminase (ADA) deficiency causes∼ 13% of cases of severe combined
immune deficiency (SCID). Treatments include enzyme replacement therapy (ERT) …

Spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID) are rare,
inherited genetic disorders with severe mortality and morbidity. The benefits of early …

Background Public health newborn screening (NBS) programs continuously evolve, taking
advantage of international shared learning. NBS for severe combined immunodeficiency …

Importance Lifelong immunoglobulin replacement therapy (IRT) is standard-of-care
treatment for congenital agammaglobulinemia but accrues high annual costs ($30 000-$90 …

Objective Inborn errors of immunity (IEI) are rare genetic conditions affecting the immune
system. The rate of IEI and their presentation, course, and treatment are all affected by a …

Background Severe combined immunodeficiency (SCID) is a syndrome characterized by
profound T-cell deficiency that is universally fatal in infancy unless immune reconstitution is …