title: Gene therapy for adenosine deaminase deficiency.
abstract: In the last decade, gene therapy for adenosine deaminase deficiency has been developed as a successful alternative strategy to allogeneic bone marrow transplant and enzyme replacement therapy. Infusion of autologous hematopoietic stem cells, corrected ex vivo by retroviral vectors and combined to low-intensity conditioning regimen, has resulted in immunologic improvement, metabolic correction, and long-term clinical benefits. These findings have opened the way to applications of gene therapy in other primary immune deficiencies using novel vector technology.
authors.affiliation: San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, via Olgettina 58, Milan 20132, Italy.
authors.forename: Barbara
authors.initials: B
authors.lastname: Cappelli
authors.forename: Alessandro
authors.initials: A
authors.lastname: Aiuti
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency; D000818:Animals; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000009:adverse effects / Q000639:trends*; D005822:Genetic Vectors; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D012190:Retroviridae / Q000235:genetics*; D016511:Severe Combined Immunodeficiency / Q000235:genetics* / Q000276:immunology / Q000628:therapy*; D019172:Transplantation Conditioning
chemical_list: D000243:Adenosine Deaminase