CFTR mRNA Based Gene Therapy for cystic fibrosis (CF)

Cystic fibrosis (CF) is a lethal autosomal recessive genetic disease which affects over 70, 000 people in the world. The responsible gene for CF was identified in 1989, so called CFTR (cystic fibrosis transmembrane conductance -regulator), and CF is caused by over 1300 mutations in CFTR. Among these mutations, deletion phenylalanine 508 (dF508) causes more than 90% cases of CF. Main pathological features are thick and dehydrate airway mucus, high sweat Cl- etc, and major site of CF pathology is lung, accompanying infection by S. aureus and P.aureginosa. Life expectancy is around 30 years. CFTR is localized in ch…