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Open AccessReview

Production and Application of Multicistronic Constructs for Various Human Disease Therapies

1
Institute of Fundamental Medicine and Biology, Kazan Federal University, 420008 Kazan, Russia
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Kazan Institute of Biochemistry and Biophysics, FRC Kazan Scientific Center of RAS, 420111 Kazan, Russia
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School of Veterinary Medicine and Science, University of Nottingham, LE12 5RD Nottingham, UK
4
Shemyakin-Ovchinnikov Institute of Bioorganic Chemistry, The Russian Academy of Sciences, 117997 Moscow, Russia
*
Author to whom correspondence should be addressed.
Pharmaceutics 2019, 11(11), 580; https://doi.org/10.3390/pharmaceutics11110580
Received: 10 October 2019 / Revised: 30 October 2019 / Accepted: 3 November 2019 / Published: 6 November 2019
(This article belongs to the Special Issue Gene Delivery Vectors and Physical Methods: Present and Future Trends)
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. View Full-Text
Keywords: 2A peptides; IRES; multicistronic constructs; gene therapy; neurodegenerative diseases; metabolic diseases; cancer; autoimmune diseases; cardiovascular diseases 2A peptides; IRES; multicistronic constructs; gene therapy; neurodegenerative diseases; metabolic diseases; cancer; autoimmune diseases; cardiovascular diseases
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Shaimardanova, A.A.; Kitaeva, K.V.; Abdrakhmanova, I.I.; Chernov, V.M.; Rutland, C.S.; Rizvanov, A.A.; Chulpanova, D.S.; Solovyeva, V.V. Production and Application of Multicistronic Constructs for Various Human Disease Therapies. Pharmaceutics 2019, 11, 580.

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