VERSIONS BASED ON ADENO-ASSOCIATED VIRUS, COMPOSITIONS AND METHODS FOR PULMONARY DELIVERY Russian patent published in 2024 - IPC C12N15/864 C07K14/47 C07K14/15 A61K48/00 A61K38/17 A61K38/16 

FIELD: chemistry; biotechnology.

SUBSTANCE: invention relates to biochemistry. Described is a method of delivering a heterologous nucleic acid into a lung cell in a subject, involving administering to the subject a composition containing a recombinant adeno-associated virus (rAAV) vector. Said vector rAAV contains (i) a capsid containing a capsid protein containing an amino acid sequence presented as SEQ ID NO: 12, or an amino acid sequence at least 90% identical to SEQ ID NO: 12 and containing Thr at amino acid 469 and Ala at amino acid 598, based on amino acid numbering presented in SEQ ID NO: 12, and (ii) a heterologous nucleic acid containing a nucleotide sequence coding a gene product. Introduction includes pulmonary, endobronchial, intranasal, intratracheal and/or intrabronchial introduction. Described is a corresponding composition containing said vector. What is also described is a method of treating cystic fibrosis in a subject, involving administering said composition to the subject. Use of said composition in preparing a medicinal agent for treating cystic fibrosis, wherein the medicinal agent is intended for pulmonary, endobronchial, intranasal, intratracheal or intrabronchial administration.

EFFECT: invention extends the range of products for treating pulmonary diseases.

30 cl, 35 dwg, 18 tbl, 7 ex