ADENO-ASSOCIATED VIRUS VECTORS FOR TREATING LYSOSOMAL STORAGE DISEASES Russian patent published in 2020 - IPC C12N15/86 C12N9/24 A61K38/47 

FIELD: medicine; pharmaceuticals.

SUBSTANCE: invention relates to adeno-associated viral vectors and pharmaceutical compositions containing them for treating lysosomal accumulation diseases, particularly for treating type IIIB mucopolysaccharidosis (MPSIIIB). Disclosed is a recombinant AAV9-vector containing a CAG promoter, SEQ ID NO: 4, associated with nucleotide sequence coding alpha-N-acetylglucosaminidase, SEQ ID NO: 1, wherein nucleotide sequence coding alpha-N-acetylglucosaminidase, SEQ ID NO: 1, is a sequence SEQ ID NO: 3, SEQ ID NO: 19 or SEQ ID NO: 22 and polyA sequence, inserted between first end repeat AAV and second end repeat AAV. What is also presented is a plasmid containing a nucleotide sequence coding alpha-N-acetylglucosaminidase, SEQ ID NO: 1. Said plasmid is either pAAV-CAG-cohNaglu with access number DSM 26626, containing the nucleotide sequence SEQ ID NO: 3, coding alpha-N-acetylglucosaminidase, SEQ ID NO: 1, or said plasmid contains nucleotide sequence SEQ ID NO: 19, coding alpha-N-acetylglucosaminidase, SEQ ID NO: 1, or said plasmid contains nucleotide sequence SEQ ID NO: 22, coding alpha-N-acetylglucosaminidase, SEQ ID NO: 1. Said vector and plasmids are used in a pharmaceutical composition, and is also used for preparing a drug for increasing activity of alpha-N-acetylglucosaminidase in the body for treating MPSIIIB or for preparing a drug for treating MPSIIIB.

EFFECT: inventions provide an effective MPSIIIB therapy.

11 cl, 24 dwg, 21 ex