MODULATION OF REP PROTEIN ACTIVITY WHEN PRODUCING CLOSED-END DNA (ceDNA) Russian patent published in 2024 - IPC C12N15/11 C12N15/86 A61K48/00 

FIELD: biotechnology.

SUBSTANCE: group of inventions including a DNA vector for delivering a polynucleotide sequence (variants), a polynucleotide for creating a DNA vector, a method of producing a DNA vector, a pharmaceutical composition for gene therapy, a kit for producing a DNA vector, a cell for producing a ceDNA vector. In one embodiment, the DNA vector is derived from a vector polynucleotide, wherein the vector polynucleotide encodes a nucleic acid sequence located between a first inverted terminal repeat (ITR) and a second ITR, wherein at least one of the first ITR and the second ITR contains a nucleic acid sequence, corresponding to a replication protein (Rep) binding site to induce replication of a DNA vector in a cell in the presence of a single type of Rep protein having at least DNA binding and DNA nicking functionality.

EFFECT: invention expands the range of means for delivering a polynucleotide sequence.

89 cl, 64 dwg, 16 tbl, 6 ex