COMPOSITIONS AND METHODS FOR CELL TRANSFECTION Russian patent published in 2024 - IPC C08G73/02 A61K31/7105 A61K38/39 A61K47/64 C12N15/87 

FIELD: chemistry.

SUBSTANCE: present invention relates to branched polymers and polyplexes, which are used in gene therapy as safe and non-toxic agents for transfection of nucleic acids. Disclosed is a polyplex containing a nucleic acid component and a highly branched poly(β-aminoester), which is a compound with structural formula

where each A is independently a linear or branched carbon chain of 1 to 30 carbon atoms, each B is independently or ; G is -C-; n is equal to at least 1; each E₁ is alkylene or heteroalkylene; each E₂ is selected from the group consisting of alkylene or heteroalkylene; each X is independently or ; each Y independently represents or ; each L is , where x is equal to 1–1000; or ; every R₁, R₂ and R₃ in each case independently represent H, C₁-C₄₀ alkyl or C₁-C₄₀ heteroalkyl, and R₁ and R₂ is unsubstituted or substituted with at least one of a hydroxyl group, an amino group or C₃-C₆ heterocyclic group; a is equal to 1–1000; b is equal to 3; c is equal to 1–3; and z is equal to 1–100; provided that at least one of R₂ and R₃ is not H. Said polyplex is used to correct the defective translation of the COL7A1 gene in treating dystrophic epidermolysis bullosa.

EFFECT: presented invention is a reliable non-viral gene delivery system for fibroblast transfection with high efficiency and safety.

43 cl, 21 dwg, 2 tbl, 10 ex