FIELD: biotechnology.
SUBSTANCE: group of inventions is described, including a lipid nanoparticle for the delivery of a non-capsid non-viral DNA vector to a target site, and a pharmaceutical composition for the delivery of heterologous nucleic acid (options). In one option of the implementation, the lipid nanoparticle contains an ionized lipid and a non-viral non-capsid DNA vector with covalently closed ends (hereinafter – ceDNA vector), where the specified ceDNA vector contains at least one heterologous nucleotide sequence functionally located between asymmetrical inverted terminal repeats (hereinafter – asymmetrical ITR), where ITR are asymmetrical relatively to each other, and where one or more of asymmetrical ITR is modified by deletion, insertion, and/or replacement in at least one of areas selected from A, A’, B, B’, C, C’, D, and D’, wherein at least one of asymmetrical ITR contains a functional site of terminal resolution and Rep binding site.
EFFECT: invention expands the arsenal of means for the delivery of a non-capsid non-viral DNA vector to a target site.
37 cl, 37 dwg, 5 tbl, 13 ex
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MODULATION OF REP PROTEIN ACTIVITY WHEN PRODUCING CLOSED-END DNA (ceDNA) | 2020 |
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Authors
Dates
2022-08-18—Published
2018-09-07—Filed