Affiliation:
1. Laboratory of Genome Editing, Research Centre for Medical Genetics, Moskvorechye 1, Moscow 115522, Russian
Federation
Abstract
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.
Publisher
Bentham Science Publishers Ltd.
Subject
Genetics (clinical),Drug Discovery,Genetics,Molecular Biology,Molecular Medicine