The first attempts at human gene therapy were made almost 10 years ago in children with a form of severe combined immunodeficiency (SCID) caused by defects in adenosine deaminase (ADA), a purine ...

PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder ... "bubble boy" disease adenosine deaminase deficiency (ADA-SCID) – originally developed by GlaxoSmithKline ...