Natural killer (NK) cells are effective in combating infections and tumors and as such are
tempting for adoptive transfer therapy. However, they are not homogeneous but can be …

The experience of the past 3 years, since the first case of leukemia was reported in a child
cured of X-linked severe combined immunodeficiency (X-SCID) by gene therapy, indicates …

Twenty-five years have passed since first attempts of gene therapy (GT) in children affected
by severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) defect …

Gene transfer into autologous hematopoietic stem cells by γ-retroviral vectors (gRV) is an
effective treatment for adenosine deaminase (ADA)–deficient severe combined …

Gene therapy with ex vivo-transduced hematopoietic stem/progenitor cells may represent a
valid therapeutic option for monogenic immunohematological disorders such as Wiskott …

Adenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction,
leading to severe combined immunodeficiency (SCID) and multiple organ damage. To …

Gene therapy clinical trials require rigorous non-clinical studies in the most relevant models
to assess the benefit-to-risk ratio. To support the clinical development of gene therapy for β …

Gene therapy for ADA-deficient SCID is an effective treatment, providing long-term clinical
benefit for affected patients. Future research will be needed to address the occurrence of …

Gene therapy is a highly attractive strategy for many types of inherited disorders of the
immune system. Adenosine deaminase (ADA) deficient-severe combined immunodeficiency …

These studies have initiated a genetic dissection of the development of human NK cells.
Further studies are warranted, including the search for genetic causes of NKD in particular …