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 

Abstract RU 2829874 C1

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

Similar patents RU2829874C1

Title Year Author Number
NEW LIVER TARGETING ADENO-ASSOCIATED VIRAL VECTORS 2019
  • Colosi, Peter Cameron
  • Ramirez, Silvia
RU2793735C2
METHODS FOR AAV DETECTION 2017
  • Jin, Xiaoying
  • O'Riordan, Catherine, R.
  • Liu, Lin
  • Zhang, Kate
RU2771622C2
TREATMENT OF COMPLEMENT-MEDIATED DISORDERS 2017
  • Lachmann, Peter
  • Alexander, Ian
RU2768982C2
VERSION OF AAV, COMPOSITIONS AND METHODS IN WHICH IT IS USED, AS WELL AS METHODS OF USING IT FOR TRANSFERRING GENES INTO CELLS, ORGANS AND TISSUES 2014
  • High, Katherine A.
  • Yazicioglu, Mustafa N.
  • Anguela, Xavier
RU2837753C2
GENOTHERAPY CONSTRUCTIONS FOR TREATMENT OF WILSON'S DISEASE 2020
  • Livingston, Christine
  • Wadsworth, Samuel
RU2807158C2
NEW CAPSID PROTEINS OF ADENO-ASSOCIATED VIRUS 2017
  • Colosi, Peter, Cameron
  • Lochrie, Michael
  • Ng, Robert
RU2779318C2
GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A 2017
  • Wang, Lili
  • Wilson, James M.
  • Sidrane, Jenny Agnes
RU2762257C2
ADENO-ASSOCIATED VIRAL VECTORS FOR TREATING MUCOLIPIDOSIS TYPE II 2016
  • Jyu, Nelson
  • Dzin, Dong-Kiu
RU2742612C2
MODIFIED FACTOR IX, AS WELL AS COMPOSITIONS, METHODS AND OPTIONS FOR THE USE OF GENE TRANSFER INTO CELLS, ORGANS AND TISSUE 2016
  • Khaj Ketrin A.
  • Angela Khaver
RU2811445C2
COMPOSITIONS AND METHODS FOR INCREASING OR ENHANCING TRANSDUCTION OF GENE THERAPY VECTORS AND FOR REMOVING OR REDUCING IMMUNOGLOBULINS 2021
  • Armour, Sean
RU2841237C1

RU 2 829 874 C1

Authors

Kotterman, Melissa

Francis, Peter

Calton, Melissa

Gonzales, Johnny

Croze, Roxanne

Schmitt, Christopher

Dates

2024-11-07Published

2021-04-26Filed