Airway Gene Therapy

摘要: Abstract Given both the accessibility and genetic basis of several pulmonary diseases, lungs airways initially seemed ideal candidates for gene therapy. Several routes access are available, many which have been refined optimized nongene drug delivery. Two respiratory cystic fibrosis (CF) α 1 ‐antitrypsin ( ‐AT) deficiency, relatively common; single responsible has identified current treatment strategies not curative. This type inherited disease was obvious initial target therapy, but it become clear that nongenetic acquired including cancer, may also be amenable to this approach. The majority preclinical clinical studies in airway involved viral vectors, although diseases such as CF, likely require repeated application, non‐viral delivery systems advantages. However, with approaches a range barriers expression limiting success alveolar region. chapter reviews these issues, aimed at overcoming them, progress into trials vectors variety diseases.