Chapter 5 - Applications of innovative gene-editing technologies in respiratory diseases

Abstract

Gene therapy is a highly promising strategy for diseases of the lung and other organs. An increased number of potential lung therapeutic targets has been reported for some genome editing technologies. Different delivery approaches such as RNA, viral, and recombinant protein are feasible in the lung since it is characteristically in a direct contact with the external environment, which facilitates easier harvesting of patient-specific pulmonary stem cells for gene-editing approaches ex vivo. Another advantage for fetal airways is their direct contact with the amniotic fluid that facilitates targeted genome editing technology approaches such as clustered regularly interspaced short palindromic repeats (CRISPR)–CRISPR-associated protein 9 (Cas9) system that is promising for inactivating or repairing disease-causing mutations. In addition, CRISPR–Cas9 system is more efficient, cheaper, faster, and more accurate than other currently existing gene-editing approaches. Several recent studies have provided proof of concept by successfully applying CRISPR/Cas9 gene-editing technology in the inactivation or repair of mutations in animal models of important human diseases. In this chapter, we will describe recent advances and potential applications of CRISPR/Cas9 genome editing technology in gene correction of important pulmonary diseases.