Perspectives on Genetic Medicine for Cystic Fibrosis

Abstract

Like any inherited protein deficiency disease, cystic fibrosis (CF) is a good candidate for gene replacement therapy. Despite the tremendous efforts of scientists worldwide invested in developing this approach, it did not lead to the expected results for various reasons discussed in this review. At the same time, the emergence of new methods of genome editing, as well as their latest modifications, makes it possible to bypass some of the problems of "classical" CF gene therapy. The review examines potential therapeutic agents for CF gene therapy, methods and routes of delivery, as well as discusses the problem of target cells for defect correction. Based on the results of these studies, editing genetic defects in the basal cells of the lungs and their counterparts in other organs will make it possible to create a drug for treating CF with a single administration.

Keywords: CFTR; Cystic fibrosis; basal cells; gene therapy; genome editing; viral vectors.