The idea of treating hereditary diseases by introducing “the right” DNA segment into the organism is not novel: it was first pronounced in the 1970s. Over the past 30 years, more than 2 thousand clinical trials of gene therapy agents have been carried out. Currently, about 50 hereditary diseases can be managed more or less effectively. In his review, Denis Rebrikov of Pirogov Russian National Research Medical University tells about the most advanced genome editing techniques, namely ZFNs, TALENs and CRISPR/Cas9. Eugenia Zotova of Marlin Biotech et al. present the results of the analysis of the efficiency of exon skipping as a method of treating muscular distrophies.