GENE THERAPY VECTORS FOR TREATING DANON'S DISEASE Russian patent published in 2024 - IPC C12N15/86 A61K48/00 

FIELD: biotechnology.

SUBSTANCE: described is a recombinant adeno-associated virus (rAAV) gene therapy vector. Virus contains polynucleotide containing 5'-IER, expressing cassette and 3'-IER, and capsid protein. Expression cassette contains a transgene coding membrane protein 2 associated with lysosomes (LAMP-2), or a functional version thereof. Expression cassette is flanked by 5'-IER and 3'-IER. Capsid protein includes capsid protein AAVrh.74 or its functional version. Capsid protein or its functional variant is characterized by at least 95% sequence identity with respect to the amino acid sequence SEQ ID NO: 2. Also described is a pharmaceutical composition containing said rAAV vector for gene therapy. Presented is a method of treating or preventing Danone's disease in a subject in need thereof, which involves administering an rAAV vector for gene therapy or a pharmaceutical composition to the subject. Also presented is a method of delivering a LAMP-2 polynucleotide coding a LAMP-2 protein to a subject and/or expressing the LAMP-2 protein in the subject, which involves administering an rAAV vector for gene therapy or a pharmaceutical composition to the subject.

EFFECT: invention extends the range of gene therapy products.

27 cl, 11 dwg, 3 tbl, 2 ex