Abstract
Gene therapy requires an effective and safe delivery vehicle for nucleic acids. In the case of non-viral vehicles, including cationic liposomes, the structure of compounds composing them determines the efficiency a lot. Currently, cationic amphiphiles are the most frequently used compounds in liposomal formulations. In their structure, which is a combination of hydrophobic and cationic domains and includes spacer groups, each component contributes to the resulting delivery efficiency. This review focuses on polycationic and disulfide amphiphiles as prospective cationic amphiphiles for gene therapy and includes a discussion of the mutual influence of structural components.
Highlights
Gene therapy is a modern and promising method for treating severe hereditary and acquired diseases, including COVID-19 immunization, through the delivery of therapeutic nucleic acids (NAs) that can replace a damaged gene, provide a new one, or block the expression of an unwanted protein [1,2]
This review focuses on polycationic and disulfide amphiphiles as prospective cationic amphiphiles for gene therapy and includes a discussion of the mutual influence of structural components
HeLa cells with complexes formed at different N/P ratios (4:1–12:1) showed showed that that liposomes provided efficient pDNA delivery exceeding that of the commercial transfectant
Summary
Gene therapy is a modern and promising method for treating severe hereditary and acquired diseases, including COVID-19 immunization, through the delivery of therapeutic nucleic acids (NAs) that can replace a damaged gene (pDNA), provide a new one, or block the expression of an unwanted protein (antisense oligonucleotides, siRNA) [1,2]. We will consider polycationic amphiphiles, of NAs intofor cells due to the formation of a system of distributed charges in the polyamine which, compared to monocationic analogs, enable the more efficient transport of NAs into matrix and their ability to facilitate NA release from endosomes. This ability is strongly cells due to the formation of a system of distributed charges in the polyamine matrix and affected by the high H+ buffer capacity of polyamines containing titratable amines results their ability to facilitate. Cl− accumulation during acidification with presumed osmotic endosome disruption and enhanced lipoplex escape [29]
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