Gene therapy of severe combined immunodeficiencies

Distribution, clinical features and treatment in Taiwanese patients with symptomatic primary immunodeficiency diseases (PIDs) in a nationwide population-based study during 1985–2010

“…Stem-cell transplantation or gene therapy (Buckley et al 1999;Fischer et al 2002;Hacein-Bey-Abina et al 2002;Thrasher and Goldblatt 2001) is used to cure severe T-cell and phagocyte defects. In seven patients with successful transplantation, cyclophosphamide and busulfan have been used to condition patients receiving bone marrow transplantation to avoid the toxic effects of total body irradiation in very young patients (Yang et al 2005).…”

Nucleic Acid Delivery and Localizing Delivery with Magnetic Nanoparticles

“…The validity of the concept and therapeutic efficacy has been demonstrated in humans using viral vectors [247]. However, this success has been overshadowed by severe adverse events [248].…”

Gene and Cell Therapy in Germany and the EU

“…Later, SCID due to a defect of the common gamma chain (gC) gene was also treated. Both deficiencies are due to autosomal recessive gene defects, leading to severe combined immunodeficiency diseases (ADA-SCID, SCID-X1) (Blaese et al, 1995;Cavazzana-Calvo et al, 2000;Fischer et al, 2002). In the recent ADA-SCID study carried out in Italy (Auti et al, 2002), patients treated with genemodified autologous haemaotopoietic stem cells gained clinical benefit.…”