title: Evaluation of ADA gene expression and transduction efficiency in ADA/SCID patients undergoing gene therapy.
abstract: A capillary electrophoresis (CE) method was developed for ADA/SCID diagnosis and monitoring of enzyme replacement therapy, as well as for exploring the transfection efficiency for different retroviral vectors in gene therapy.
authors.affiliation: Dipartimento di Medicina Interna, Scienze Endocrino-Metaboliche e Biochimica, Università di Siena, Siena, Italy.
authors.forename: F
authors.initials: F
authors.lastname: Carlucci
authors.forename: A
authors.initials: A
authors.lastname: Tabucchi
authors.forename: A
authors.initials: A
authors.lastname: Aiuti
authors.forename: F
authors.initials: F
authors.lastname: Rosi
authors.forename: F
authors.initials: F
authors.lastname: Floccari
authors.forename: R
authors.initials: R
authors.lastname: Pagani
authors.forename: E
authors.initials: E
authors.lastname: Marinello
mesh_terms: D000243:Adenosine Deaminase / Q000096:biosynthesis* / Q000172:deficiency* / Q000235:genetics*; D018952:Antigens, CD34 / Q000096:biosynthesis; D002851:Chromatography, High Pressure Liquid; D019075:Electrophoresis, Capillary; D004912:Erythrocytes / Q000378:metabolism; D005786:Gene Expression Regulation; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D012190:Retroviridae / Q000235:genetics; D016511:Severe Combined Immunodeficiency / Q000235:genetics* / Q000628:therapy*; D013053:Spectrophotometry; D013601:T-Lymphocytes / Q000378:metabolism; D013997:Time Factors; D014162:Transfection
chemical_list: D018952:Antigens, CD34; D000243:Adenosine Deaminase