Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …

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

For over two decades, the EBMT has updated recommendations on indications for
haematopoietic cell transplantation (HCT) practice based on clinical and scientific …

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 …

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

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 Severe combined immunodeficiency due to adenosine deaminase (ADA)
deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency. Methods We …

Chronic granulomatous disease (CGD) is a primary immunodeficiency of phagocyte function
due to defective NADPH oxidase (phox). Compared with the common types of CYBB/gp91 …

Despite the ups and downs in the field over three decades, the science of gene therapy has
continued to advance and provide enduring treatments for increasing number of diseases …

Chronic granulomatous disease (CGD) is a primary immunodeficiency resulting in life-
threatening infections and inflammatory complications. Allogeneic hematopoietic cell …