VECTORS CONTAINING SPACER/FILLER POLYNUCLEOTIDE SEQUENCES, AND METHODS OF USING THEM Russian patent published in 2020 - IPC C12N15/00 C12N15/86 A61K39/23 

FIELD: biotechnology.

SUBSTANCE: described is a recombinant vector plasmid for transferring and delivering a heterogeneous polynucleotide sequence into a mammalian cell, comprising: an adeno-associated virus (AAV) vector genome containing a heterologous polynucleotide sequence and an expression control sequence, which triggers a transcription of the heterologous polynucleotide sequence; and main sequence, containing a first inert polynucleotide filler sequence or a spacer polynucleotide sequence, wherein said heterologous polynucleotide sequence in length is less than 4.7 Kb and is located between two sequences of inverted end repeat (ITR) adenoassociated virus (AAV), wherein said first inert polynucleotide filler sequence or spacer polynucleotide sequence is located outside the ITR AAV and has length of 4.5–10 Kb or 6–8 Kb and where length of said main sequence is at least 7.0 Kb. Said heterologous polynucleotide codes a protein which provides therapeutic benefit, or encodes an inhibitory nucleic acid, which reduces or inhibits expression of an undesired or defective gene.

EFFECT: invention reduces impurities of residual plasmid DNA.

42 cl, 6 dwg, 3 ex