Key result
Multicistronic vectors using IRES and 2A peptides enable joint multigene delivery for gene therapy.
Why the study?
Fundamental issues in molecular and cellular biology require simultaneous delivery and joint expression of several genes for the treatment and modeling of human diseases.
Multicistronic gene therapy vectors offer a promising platform for complex multigenic disease treatment, though vector optimization is still required to improve translational efficiency.
Multicistronic vectors may aid multigenic disease modeling and therapy; leaves open optimal design for clinical translation.
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.
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Shaimardanova et al. (2019) conducted a review in Various human diseases (neurodegenerative, metabolic, autoimmune, cardiovascular, oncological, infectious). Multicistronic vectors (IRES and self-cleaving 2A peptides) was evaluated. Multicistronic vectors based on internal ribosome entry sites (IRES) and self-cleaving 2A peptides enable simultaneous delivery and joint expression of multiple genes for disease modeling and gene therapy.
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