GENE THERAPY OF CHRONIC GRANULOMATOUS DISEASE

摘要

The present invention relates to the field of gene therapy. In particular, it provides a nucleic acid encoding gp91phox, wherein codon-usage of said gp91phox nucleic acid is optimized for expression in human cells, and an expression construct, in particular a retroviral vector suitable for gene therapy of chronic granulomatous disease, comprising said nucleic acid. Additionally, methods of preparing cells comprising the construct, said cells and pharmaceutical compositions comprising the construct or cells of the invention are provided.