The field of gene therapy is striving more than ever to define a path to the clinic and the
market. Twenty gene therapy products have already been approved and over two thousand …

In 2000, the National Institute of Health launched the National Nanotechnology Initiative to
support, coordinate, and advance research and development of nanoscale projects. The …

Adenosine deaminase (ADA) deficiency leads to an accumulation of toxic purine
degradation by-products, most potently affecting lymphocytes, leading to adenosine …

Pioneering gene therapy trials have shown that the genetic engineering of haematopoietic
stem and progenitor cells can be an alternative to allogeneic transplantation in the treatment …

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 therapy is an innovative treatment for Primary Immune Deficiencies (PIDs) that uses
autologous hematopoietic stem cell transplantation to deliver stem cells with added or edited …

Deficiency of adenosine deaminase (ADA, EC3. 5.4. 4), a housekeeping enzyme of purine
metabolism encoded by the Ada gene, is a cause of human severe combined immune …

Severe combined immunodeficiencies (SCIDs) are a group of inherited disorders
responsible for severe dysfunctions of the immune system. These diseases are life …

Patients lacking functional adenosine deaminase activity have severe combined
immunodeficiency (ADA SCID), which can be treated with ADA enzyme replacement therapy …

Inborn errors of immunity (IEI) are a heterogenous group of disorders driven by genetic
defects that functionally impact the development and/or function of the innate and/or …