VIRIONS OF AN ADENO-ASSOCIATED VIRUS WITH A VARIANT CAPSID AND METHODS FOR USING THEM Russian patent published in 2021 - IPC A61K39/235 A61K48/00 C12N15/864 

FIELD: biotechnology.

SUBSTANCE: described is a virion of recombinant adeno-associated rAAV virus containing: a) variant AAV capsid protein, which contains a peptide insert compared to the corresponding capsid protein of the parent AAV, where the peptide insert has length of 7 amino acids to 10 amino acids, where the insertion site is located between two adjacent amino acids in the position between the amino acids corresponding to amino acids 570 and 611 VP1 of AAV2 or corresponding position in the capsid protein of the other AAV serotype; and b) a heterologous nucleic acid containing a nucleotide sequence encoding a gene product. Disclosed is a pharmaceutical composition containing said virion. Disclosed is a method of treating retinal disease, which involves administering to an individual in need thereof an effective amount of the described virion.

EFFECT: implementing the invention provides higher infectivity of the retinal cell, since AAV virions are introduced by intravitreal injection compared to wild-type AAV.

15 cl, 2 tbl, 3 ex, 19 dwg