ABSTRACT
Genome editing is a type of DNA engineering which allows precise modifications in the genomic DNA. Genome editing is mediated through programmable nucleases which recognize and bind specific sequences in the genome. Zinc-finger nucleases were discovered as one of the first useful tools for genome editing. Genetic lung diseases are devastating disorders, usually resulting in high morbidity and mortality. Most lung gene therapies used delivery route via the airways, including nebulization, intratracheal administration, and bronchoscopy. The potential to provide long-term effect is a great advantage of therapeutic genome editing over small molecule drugs. Off-target gene modification is one of the key concerns for genome editing therapy. Many studies investigated on how to precisely measure the frequencies and locations of off-target sites, and how to minimize the off-target effects. Besides off-target, the genome editing nucleases may induce other unexpected side effects. Genome editing targets rare or ultra-rare lung genetic diseases.
