Careful adjustment of Epo non-viral gene therapy for β-thalassemic anaemia treatment

摘要: Background In situ production of a secreted therapeutic protein is one the major gene therapy applications. Nevertheless, plasmatic secretion peak transgenic may be deleterious in many applications including Epo therapy. transfer appears to promising alternative recombinant for severe anaemia treatment despite polycythemia was reached previous studies. Therefore, an accurate level transgene expression required application safety. The aim this study adapt posology and administration schedule chosen avoid potentially toxic maintain efficiency. potential repeated muscular electrotransfer light Epo-plasmid doses evaluated β-thalassemic mice.