VECTORS BASED ON ADENO-ASSOCIATED VIRUSES FOR TREATING MUCOPOLYSACCHARIDOSES Russian patent published in 2021 - IPC A61K48/00 C12N15/864 C12N9/16 

FIELD: biotechnology; pharmaceutics.

SUBSTANCE: recovered nucleic acid has a nucleotide sequence encoding an iduronate-2-sulfatase protein presented in SEQ ID NO: 1, wherein said nucleotide sequence is selected from SEQ ID NO: 5 and SEQ ID NO: 8, for use as a drug for treating type II mucopolysaccharidosis. There are presented versions of plasmids, a recombinant vector based on adeno-associated serotype 9 virus for use as a drug for treating type II mucopolysaccharidosis. Pharmaceutical composition contains a therapeutically effective amount of nucleic acid, a plasmid or a recombinant vector for use as a therapeutic agent for type II mucopolysaccharidosis. Method of producing vectors involves the following steps. Providing a first vector comprising a nucleotide sequence selected from SEQ ID NO: 5 and SEQ ID NO: 8, located between the first end repeat AAV and the second end repeat AAV, a CAG promoter operably linked to a nucleotide sequence; a second vector comprising AAV rep-gene and AAV cap-gene from serotype 9; and a third vector containing a helper functional element adenovirus gene. Competent co-transfection of competent cells with said vectors is performed. Transfected cells are cultured and expression vectors are purified from the culture. Method of producing the plasmid involves the following steps. Nucleotide sequence is excised from the initial plasmid by digestion with Mlul/EcoRI. Nucleotide sequence selected from SEQ ID NO: 5 and SEQ ID NO: 8 is cloned between two restriction sites of a basic plasmid based on AAV pAAV-CAG. Isolated cell contains nucleic acid for treating type II mucopolysaccharidosis.

EFFECT: group of inventions provides new sequences which are used for treating type II mucopolysaccharidosis.

13 cl, 14 ex, 47 dwg