Genome editing and gene therapy methods in the treatment of human diseases

S.N. Ptitsina

All-Russian Institute of Scientific and Technical Information of the Russian Academy of Sciences, Moscow

This review is devoted to one of the urgent problems of modern biomedicine — gene therapy of hereditary human diseases using new genetic technologies (genome editing systems, ZFN and TALEN nucleases, CRISPR-Cas9). The information from the databases of All-Russian Institute of Scientific and Technical Information of the Russian Academy of Sciences, SCOPUS, PubMed for 2015–2020 is analyzed. It is concerned both the general issues description of the new biotechnologies and disease models development, the use of various vectors, delivery systems, genetic engineering methods, and the successful application of the above technologies in the treatment of specific diseases. The article presents examples of modeling and therapy of neuromuscular, oncological, immunological, hematological and neurodegenerative human diseases. It also discusses the ethical issues that inevitably arise when editing the human genome. The results of the publication analysis from databases (All-Russian Institute of Scientific and Technical Information of the Russian Academy of Sciences, SCOPUS, PubMed for 2015–2020) provide an opportunity to generate new scientific and information products on the problems of medical genetics. It is expected that the data obtained will be useful to both geneticists and clinicians.

Keywords: hereditary diseases, biomedicine, genetics, gene therapy, methods, genome editing, CRISPR-Cas9 system, biotechnologies, databases.

For citation: Ptitsina S.N. Genome editing and gene therapy methods in the treatment of human diseases. RMJ. 2021;10:57–62.