ADENO-ASSOCIATED VIRAL VECTORS FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSIS TYPE IV A Russian patent published in 2023 - IPC C12N9/16 C12N15/864 

FIELD: biotechnology.

SUBSTANCE: isolated nucleic acid is described with a nucleotide sequence from 75% to 90% identical to the nucleotide sequence as shown in SEQ ID NO: 1, wherein the said nucleic acid encodes a functional human N-acetyl-galactosamine-6-sulfatase, and wherein the said nucleic acid is selected from the group consisting of SEQ ID NO: 3, SEQID NO: 5 and SEQ ID NO: 7, for use in the treatment of mucopolysaccharidosis type IVA or Morquio A syndrome. Also an expression vector containing a nucleic acid, the said nucleic acid, for use in the treatment of mucopolysaccharidosis type IVA or Morquio A syndrome is provided. In addition, a pharmaceutical composition containing a therapeutically effective amount a nucleic acid or vector for use in the treatment of mucopolysaccharidosis type IVA or Morquio A syndrome is described. The use of any vector in the manufacture of a medicament is described. A method for obtaining a vector is described including the following stages: (i) providing a cell containing a nucleic acid according to claim 1, AAV Cap proteins, AAV Rep proteins, and optionally viral proteins on which AAV replication depends, (ii) maintaining the cell under conditions suitable for assembly of AAV, and (iii) purification of the adeno-associated viral vector produced by the cell.

EFFECT: invention expands the arsenal of agents for the treatment of mucopolysaccharidosis type IVA or Morquio A syndrome.

13 cl, 14 dwg, 15 ex