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Production and Application of Multicistronic Constructs for Various Human Disease Therapies

Shaimardanova, Alisa A.; Kitaeva, Kristina V.; Abdrakhmanova, Ilmira I.; Chernov, Vladislav M.; Rutland, Catrin S.; Rizvanov, Albert A.; Chulpanova, Daria S.; Solovyeva, Valeriya V.

Authors

Alisa A. Shaimardanova

Kristina V. Kitaeva

Ilmira I. Abdrakhmanova

Vladislav M. Chernov

Catrin S. Rutland

Albert A. Rizvanov

Daria S. Chulpanova

Valeriya V. Solovyeva



Abstract

The development of multicistronic vectors has opened up new opportunities to address the fundamental issues of molecular and cellular biology related to the need for the simultaneous delivery and joint expression of several genes. To date, the examples of the successful use of multicistronic vectors have been described for the development of new methods of treatment of various human diseases, including cardiovascular, oncological, metabolic, autoimmune, and neurodegenerative disorders. The safety and effectiveness of the joint delivery of therapeutic genes in multicistronic vectors based on the internal ribosome entry site (IRES) and self-cleaving 2A peptides have been shown in both in vitro and in vivo experiments as well as in clinical trials. Co-expression of several genes in one vector has also been used to create animal models of various inherited diseases which are caused by mutations in several genes. Multicistronic vectors provide expression of all mutant genes, which allows the most complete mimicking disease pathogenesis. This review comprehensively discusses multicistronic vectors based on IRES nucleotide sequence and self-cleaving 2A peptides, including its features and possible application for the treatment and modeling of various human diseases.

Journal Article Type Article
Publication Date Nov 6, 2019
Journal Pharmaceutics
Publisher MDPI
Peer Reviewed Peer Reviewed
Volume 11
Pages 580
Institution Citation Shaimardanova, A. A., Kitaeva, K. V., Abdrakhmanova, I. I., Chernov, V. M., Rutland, C. S., Rizvanov, A. A., …Solovyeva, V. V. (2019). Production and Application of Multicistronic Constructs for Various Human Disease Therapies. Pharmaceutics, 11, 580. https://doi.org/10.3390/pharmaceutics11110580
DOI https://doi.org/10.3390/pharmaceutics11110580
Keywords 2A peptides; IRES; Multicistronic constructs; Gene therapy; Neurodegenerative diseases; Metabolic diseases; Cancer; Autoimmune diseases; Cardiovascular diseases
Publisher URL https://www.mdpi.com/1999-4923/11/11/580

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