FIELD: medicine.
SUBSTANCE: invention relates to a composition for the delivery of nucleic acids. The composition contains (a) a cationic peptide or polymer; (b) a cationic or permanently cationic lipidoid compound; and (c) a nucleic acid compound. In this case, the specified cationic or permanently cationic lipidoid compound contains a central nitrogen atom connected with С2-3 alkylene groups with three groups of Formula IIa and/or Formula IIb: -N(R1)-CH2-CH(R5)-R2 (Formula IIa), -N+(R3)(R4)-CH2-CH(R5)-R2 (Formula IIb), and independently for each individual group of Formula IIa or Formula IIb R1 is selected from hydrogen or C1-C4-alkyl, R2 is selected from linear C6, С8 or С10-alkyl or branched, saturated, or unsaturated C6-C16 hydrocarbyl chain, R3 and R4 are selected from C1-C4-alkyl and R5 is a hydrogen or hydroxyl. The specified cationic peptide or polymer is a compound containing at least one cationic group P, containing at least one group-SH, capable of forming a disulfide bond, or its disulfide-coupled multimer. Group P is selected from a polymer group having a molecular weight of 0.5 kDa to 30 kDa, or a peptide group consisting of 3-100 amino acids, with at least 10% of the total amino acids of the specified peptide group being the main amino acids selected from Arg, Lys, His and/or Orn. The invention also relates to nanoparticles for the delivery of nucleic acids containing the specified composition, a set for the preparation of the composition, a vaccine and a method of delivery and RNA to the eye.
EFFECT: invention provides high efficiency of the composition, and ability to deliver nucleic acids to mammalian cells.
34 cl, 11 dwg, 8 tbl, 11 ex
Authors
Dates
2021-07-07—Published
2017-06-09—Filed