Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

Scientists treated 50 patients with Severe combined immunodeficiency due to adenosine deaminase (ADA) with an investigational gene therapy composed of autologous CD34⁺ hematopoietic stem and progenitor cells transduced ex vivo with a self-inactivating lentiviral vector encoding human ADA.