Evaluation of ADA gene expression and transduction efficiency in ADA/SCID patients undergoing gene therapy

Nucleosides Nucleotides Nucleic Acids. 2004 Oct;23(8-9):1245-8. doi: 10.1081/NCN-200027508.

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.

MeSH terms

  • Adenosine Deaminase / biosynthesis*
  • Adenosine Deaminase / deficiency*
  • Adenosine Deaminase / genetics*
  • Antigens, CD34 / biosynthesis
  • Chromatography, High Pressure Liquid
  • Electrophoresis, Capillary
  • Erythrocytes / metabolism
  • Gene Expression Regulation*
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Humans
  • Retroviridae / genetics
  • Severe Combined Immunodeficiency / genetics*
  • Severe Combined Immunodeficiency / therapy*
  • Spectrophotometry
  • T-Lymphocytes / metabolism
  • Time Factors
  • Transfection

Substances

  • Antigens, CD34
  • Adenosine Deaminase