title: Drug evaluation: ADA-transduced hematopoietic stem cell therapy for ADA-SCID.
abstract: San Raffaele Telethon Institute for Gene Therapy is developing an adenosine deaminase-transduced hematopoietic stem cell therapy for the potential intravenous treatment of adenosine deaminase deficiency in severe combined immunocompromised individuals.
authors.affiliation: National Neuroscience Institute, National University of Singapore, Nanyang Technological University, 11 Jalan Tan Tock Seng, 308433 Singapore. obgpjt@nus.edu.sg
authors.forename: Phillippe
authors.initials: P
authors.lastname: Taupin
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency / Q000235:genetics / Q000378:metabolism*; D000818:Animals; D002478:Cells, Cultured; D002986:Clinical Trials as Topic; D004353:Drug Evaluation, Preclinical; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006412:Hematopoietic Stem Cells / Q000378:metabolism*; D006801:Humans; D016511:Severe Combined Immunodeficiency / Q000201:enzymology / Q000235:genetics / Q000628:therapy*; D019172:Transplantation Conditioning
chemical_list: D000243:Adenosine Deaminase