期刊
JOURNAL OF DRUG TARGETING
卷 21, 期 10, 页码 914-925出版社
TAYLOR & FRANCIS LTD
DOI: 10.3109/1061186X.2013.829078
关键词
Cystic fibrosis transmembrane conductance regulator; gene therapy; nanoparticles; pulmonary delivery; viral and non-viral delivery
资金
- National Institutes of Health [R01 CA111766, R01 CA138533]
Cystic fibrosis (CF) is an autosomal recessive monogenetic disease that afflicts nearly 70 000 patients worldwide. The mutation results in the accumulation of viscous mucus in multiple organs especially in the lungs, liver and pancreas. High associated morbidity and mortality is caused by CF due to the lack of effective therapies. It is widely accepted that morbidity and mortality caused by CF is primarily due to the respiratory manifestations of the disease. Consequently, several approaches were recently developed for treatment of lung complications of CF. However, the lack of effective methods for delivery and especially targeted delivery of therapeutics specifically to lung tissues and cells limits the efficiency of the therapy. Local pulmonary delivery of therapeutics has two major advantages over systemic application. First, it enhances the accumulation of therapeutics specifically in the lungs and therefore increases the efficiency of the treatment. Second, local lung delivery substantially prevents the penetration of the delivered drug into the systemic circulation limiting adverse side effects of the treatment on other organs and tissues. This review is focused on different approaches to the treatment of respiratory manifestations of CF as well as on methods of pulmonary delivery of therapeutics.
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