Evaluation of ADA gene expression and transduction efficiency in ADA/SCID patients undergoing gene therapy.
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.
Carlucci
F
F
Dipartimento di Medicina Interna, Scienze Endocrino-Metaboliche e Biochimica, Università di Siena, Siena, Italy.
Tabucchi
A
A
Aiuti
A
A
Rosi
F
F
Floccari
F
F
Pagani
R
R
Marinello
E
E
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
D018952:Antigens, CD34; D000243:Adenosine Deaminase